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Highmark Health Blog | Gene Therapy Research: Dr. Passineau

Michael Passineau, PhD, is a man who speaks in metaphors. For good reason he works within a realm of medicine not many people understand. Director of the Gene Therapy Program at Allegheny Health Network (AHN) and a leading force behind AHNs scientific research to address clinical needs, Passineau leans into the way a good metaphor can bring clarity to challenging conceptsincluding the nature of his work.

I think of clinicians as chefs, he says. At the end of each day, theyve done something tangible. Theyve made a meal. Researchers, on the other hand, are a bit like sculptors. We can work for years on something, but once its complete, its permanent.

For over a decade, his research has revolved around gene therapy more specifically, the use of ultrasound technology, instead of viral administration, to deliver therapeutic DNA into the cells of salivary glands. The goal: restore saliva flow to patients who suffer from radiation-induced dry mouth, or xerostomia.

Supported by grants from the National Institutes of Health (NIH), Passineau, radiation oncologist Dr. Mark Trombetta, and their research team are on track to petition the U.S Food and Drug Administration for Investigational New Drug (IND) status, which would allow their work to move out of the lab and into Phase 1 clinical trials with humans.

Xerostomia is an iatrogenic complication, meaning it is caused by treatment in this case, head and neck radiation to treat cancer. When the beam of radiation passes through the head, it damages the salivary glands, resulting in chronic dry mouth. This can lead to permanent loss of function in the salivary glands, difficulty eating, and loss of teeth.

Its anything but trivial, says Passineau. To illustrate how this condition impacts a persons quality of life, I often have donors and executives take a piece of surgical gauze and chew on it while I describe my research. After about five minutes, they understand how difficult it really is.

While there are a few existing medications used to treat xerostomia, they are difficult to administer, and their effects are not long lasting. Most people deal with the condition by carrying a water bottle at all times or by taking saliva substitutes. Unfortunately, these options dont work particularly well.

Were all made of trillions of cells, says Passineau, beginning an attempt to explain gene therapy in a nutshell.

Each cell has a role to play, whether its beating heart muscle, growing hair and nails, or perceiving light signals in your retina, he continues. The way those biochemical machines are engineered is dictated by your genomic DNA, which is DNA you get from your parents. Those genes code for proteins, which are the gears and springs that make cells function as biochemical machines.

Gene therapy means reprogramming the cell changing the machine code telling the cell how to work. That requires getting new DNA to travel inside the cell. Passineau says this is one of the most difficult tasks in the world since our cells are designed to repel foreign DNA.

In nature, foreign DNA gets into human cells only through viral infection or during conception. Virally administered gene therapy approaches have been developed, but one drawback is that after a viral vector is introduced into the body, the immune system fights back and will also react to the vector on subsequent treatments, making them ineffective.

Thats where Passineaus research comes in. Weve developed a method of delivering genes that doesnt require viral administration, he explains. Instead, we use soundwaves.

To understand how ultrasonic administration, or sonoporation, works, Passineau turns again to metaphor.

Picture an agricultural pond, with a thick layer of algae on it. If you throw a ping-pong ball into the middle, it will just sit on top, he says. That is very much what a cell membrane is like the outer covering is rather rigid. So, to deliver the genes, we have to get through the cell membrane. It is only seven nanometers thick but its the longest seven nanometers in nature for someone like me.

Passineaus ground-breaking research uses soundwaves to temporarily alter the permeability of the cell membrane, allowing for the transfer of therapeutic DNA into the cell.

Lets understand how this works in our pond metaphor before getting into what that means for gene therapy.

Imagine we explode a grenade above the pond, Passineau says. For a moment, the layer of slime would open up, and youd see down to the bottom of the pond. Then, it would close again.

In Passineaus lab, the grenade is a mix of a gene drug for xerostomia known as Aquaporin-1 and a solution of microbubbles. Used routinely in cardiac imaging and other medical applications, microbubbles have a resonant frequency that can be used to create the desired explosion.

The classic example is a crystal glass if an opera singer hits the right frequency for that glass, it will vibrate. If she really turns up the volume, the glass will shatter, because it cant absorb the energy, Passineau says. That same thing happens with the microbubbles.

So after administering the microbubble and Aquaporin-1 solution to the treatment site, a low-frequency ultrasound beam is used to create an ultrasonic acoustic field in which the bubbles vibrate. Turn up the power, and the bubbles implode. That opens up the cell membrane long enough for the gene drug to get in, before it closes back up.

For gene therapy researchers like Passineau, the membrane around our cells is the longest seven nanometers in nature.

Sonoporation works well for what were doing in the salivary glands, but not so well for the heart, and certainly not for the brain, Passineau points out. However, we have other applications we intend to use this research for.

He explains that one promising use involves Sjogrens syndrome, an autoimmune disease affecting nearly 4 million Americans (90 percent of whom are women). The diseases debilitating symptoms include severe dry mouth, which may be treatable with Passineaus gene therapy technique.

Another research area he says he is excited about is the use of gene therapy to combat obesity and overeating. Do you remember when you were a kid and youd eat too fast and your mom would tell you to slow down because your brain didnt know whether or not your stomach was full yet? he asks. Well, that was absolutely true.

He explains that, when we eat, our intestines stretch and release a protein called peptide YY (PYY), which circulates through the blood, eventually entering the saliva and interacting with receptors on your tongue.

Think of your appetite as a glass of water, he says. To feel full, you have to fill the glass with PYY. Some people have bigger glasses than others, but if we can use gene therapy to modify saliva and make the glass start half full, then a person would feel full without needing to eat as much.

Passineau adds that poor health outcomes and high costs associated with obesity make this an attractive target for research investment. Obesity adds billions of dollars to the cost of medical care in the U.S. each year, and some studies estimate the cost as high as $190 billion per year.

If gene therapy was this easy, everyone would be doing it. Instead, as Passineau points out, it is one of the most difficult tasks in the world.

At AHN, research is a small but important piece of the operation, Passineau says. Its important to note that everything we do in research is driven by physicians who have recognized clinical needs, and who have partnered with us to develop novel solutions.

Similarly, looking at the value that research can deliver, and its potential impact on both health and overall health care costs, Passineau says that federal funding has an essential role in advancing further discoveries in areas like gene therapy and sonoporation. Government investment really is the lifeblood of what drives research, he says.

Another impact on the success and pace of advances in medical research is whether talented, driven young people decide to take this path. Passineau admits that, like the process of research itself, the path to becoming a successful researcher can be long and sometimes feels like three steps forward, two steps back. But if the work feels meaningful, its all worth it.

I landed where I am today because I figured out what I was good at, he says. Inventing, solving big picture problems and helping people.

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Gene Therapy Questions | FAQs – Dana-Farber/Boston …

Frequently Asked QuestionsWhatis gene therapy?

Some diseases are caused by errors (mutations) inspecific genes. Gene therapy delivers DNA into cells to replacemutated (bad) or missing genes or to add new, good genes.

Scientists are investigating a number of differentways to do this. Right now, gene therapy is only done through research studiescalled clinical trials. Unlike medicine, gene therapy directly addresses the underlyinggenetic problem, not just the symptoms.

Genes are in the nucleus of every living cell. A gene is an instruction manual for the body. Itgives the direction to make the proteins that make the body work.

A gene cannot be inserted directly into a cell.Instead, a carrier called a vector is genetically engineered to deliver thegene. Viruses are usually used as the vectors because they are very good atinfecting cells and inserting the gene(s) into the cells DNA. Types of viralvectors are retrovirus, adenovirus, adeno-associated virus and herpes simplexvirus.

No. The virus is specially engineered to remove the infectious piece. We only keep the part of the virus that is good at burrowinginto a cells nucleus. Once the virus delivers the gene into the cell, thevirus slips away.

It is not for all genetic diseases. It is only forsome diseases caused by a single gene mutation. Some diseases that might betreated with gene therapy are:

The goal is to cure a disease or make changes so thebody can better fight off disease. It does not correct 100% of your childs cells.Instead, every time a cell with the good gene reproduces, it carries a copyof the new healthy gene.

The vector can be injected or given by IV directlyinto a specific tissue. Or a sample of cells can be removed and exposed to thevector in a laboratory. The cells with the vector are then returned to thepatient.

1) Stem cells are collected in one of two ways: by bone marrow aspiration, or by purifying blood drawn through a central line in a process called apheresis.

2) Before the infusion, most children have chemotherapy. This makes room for the new cells by getting rid of the existing cells in the bone marrow.

3) In the laboratory, the stem cells from the blood or bone marrow are exposed to a virus or other type of vector containing the desired genes.

4) Once the stem cells take up the vector and merge the genes into cells DNA, the cells are given back to the patient in an IV infusion.

Bone marrow transplants usestem cells from another person (a donor). Gene therapy uses your childs owncells. Using your childs own cells is a benefit because there is no risk ofrejection, or graft vs. host disease, like there is with donor cells. Genetherapy is still only offered through clinical trials and at only a fewresearch hospitals and centers.

Gene therapy is still very new,and is mostly used to treat children who cannot be cured by standardtreatments. Gene therapy is not for everydisease or a good fit for every patient. Your childneeds to meet certain criteria for safety reasons. Your childs doctor willtalk to you about whether your child is a good fit for a gene therapy clinicaltrial.

Your child will have 410 daysof chemotherapy before the infusion. This is called chemotherapy conditioning.It clears out bone marrow to make room for the new stem cells. This has typicalside effects from chemotherapy, like nausea/vomiting, mouth sores and pain.

Your child has the transfusionon the Bone Marrow Transplant floor (6 West) at the Jimmy Fund Clinic. It is given one timeintravenously (through an IV), just like a blood transfusion. It takes 1530minutes. The amount of time your childwill stay in the hospital depends on many factors. Most children stay 46weeks.

Your child will have bloodtests to check for the vector in the cells, and to see how the cells are responding. Your child will come in forfollow-ups frequently. Your childs care team will talk to you about when youshould call your childs doctor. Always call with questions or concerns or ifyou notice signs of an infection.

Many research studies areunderway to test gene therapy as a safe treatment for a growing number ofdiseases. Improvements have already beenmade in safety. Early gene therapy trials showed a high risk of turning ononcogenes that cause cancer. Now, experts have retooled the vector to lower thelikelihood of turning on oncogenes.

Learn more

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CRISPR gene editing – Wikipedia

Gene editing method

CRISPR gene editing is a method by which the genomes of living organisms may be edited. It is based on a simplified version of the bacterial CRISPR/Cas (CRISPR-Cas9) antiviral defense system. By delivering the Cas9 nuclease complexed with a synthetic guide RNA (gRNA) into a cell, the cell's genome can be cut at a desired location, allowing existing genes to be removed and/or new ones added.[1] The Cas9-gRNA complex corresponds with the CAS III CRISPR-RNA complex in the accompanying diagram.

While genomic editing in eukaryotic cells has been possible using various methods since the 1980s, the methods employed had proved to be inefficient and impractical to implement on a larger scale. Genomic editing leads to irreversible changes to the gene. Working like genetic scissors, the Cas9 nuclease opens both strands of the targeted sequence of DNA to introduce the modification by one of two methods. Knock-in mutations, facilitated via Homology Directed Repair (HDR), is the traditional pathway of targeted genomic editing approaches.[2] This allows for the introduction of targeted DNA damage and repair. HDR employs the use of similar DNA sequences to drive the repair of the break via the incorporation of exogenous DNA to function as the repair template.[2] This method relies on the periodic and isolated occurrence of DNA damage at the target site in order for a repair to commence. Knock-out mutations caused by Cas9/CRISPR results in the repair of the double-strand break by means of NHEJ (Non-Homologous End Joining). NHEJ can often result in random deletions or insertions at the repair site disrupting or altering gene functionality. Therefore, genomic engineering by CRISPR-Cas9 allows researchers the ability to generate targeted random gene disruption.

Because of this, the precision of genomic editing is a great concern. With the discovery of CRISPR and specifically the Cas9 nuclease molecule, efficient and highly selective editing is now a reality. Cas9 allows for a reliable method of creating a targeted break at a specific location as designated by the crRNA and tracrRna guide strands.[3] Cas9 derived from Streptococcus pyogenes bacteria has facilitated the targeted genomic modification in eukaryotic cells. The ease with which researchers can insert Cas9 and template RNA in order to silence or cause point mutations on specific loci has proved invaluable to the quick and efficient mapping of genomic models and biological processes associated with various genes in a variety of eukaryotes. A newly engineered variant of the Cas9 nuclease has been developed that significantly reduces off-target manipulation. Called spCas9-HF1 (Streptococcus pyogenes Cas9 High Fidelity 1), it has a success rate of modification in vivo of 85% and undetectable off-target manipulations as measured by genome wide break capture and targeted sequencing methods used to measure total genomic changes.[4][5]

CRISPR-Cas genome editing techniques have many potential applications, including medicine and crop seed enhancement. The use of CRISPR-Cas9-gRNA complex for genome editing[6] was the AAAS's choice for breakthrough of the year in 2015.[7] Bioethical concerns have been raised about the prospect of using CRISPR for germline editing.[8]

In the early 2000s, researchers developed zinc finger nucleases (ZFNs), synthetic proteins whose DNA-binding domains enable them to create double-stranded breaks in DNA at specific points. In 2010, synthetic nucleases called transcription activator-like effector nucleases (TALENs) provided an easier way to target a double-stranded break to a specific location on the DNA strand. Both zinc finger nucleases and TALENs require the creation of a custom protein for each targeted DNA sequence, which is a more difficult and time-consuming process than that for guide RNAs. CRISPRs are much easier to design because the process requires making only a short RNA sequence.[9]

Whereas RNA interference (RNAi) does not fully suppress gene function, CRISPR, ZFNs and TALENs provide full irreversible gene knockout.[10] CRISPR can also target several DNA sites simultaneously by simply introducing different gRNAs. In addition, CRISPR costs are relatively low.[10][11][12]

CRISPR-Cas9 genome editing is carried out with a Type II CRISPR system. When utilized for genome editing, this system includes Cas9, crRNA, tracrRNA along with an optional section of DNA repair template that is utilized in either non-homologous end joining (NHEJ) or homology directed repair (HDR).

CRISPR-Cas9 often employs a plasmid to transfect the target cells.[13] The main components of this plasmid are displayed in the image and listed in the table. The crRNA needs to be designed for each application as this is the sequence that Cas9 uses to identify and directly bind to the cell's DNA. The crRNA must bind only where editing is desired. The repair template is designed for each application, as it must overlap with the sequences on either side of the cut and code for the insertion sequence.

Multiple crRNAs and the tracrRNA can be packaged together to form a single-guide RNA (sgRNA).[14] This sgRNA can be joined together with the Cas9 gene and made into a plasmid in order to be transfected into cells.

CRISPR-Cas9 offers a high degree of fidelity and relatively simple construction. It depends on two factors for its specificity: the target sequence and the PAM. The target sequence is 20 bases long as part of each CRISPR locus in the crRNA array.[13] A typical crRNA array has multiple unique target sequences. Cas9 proteins select the correct location on the host's genome by utilizing the sequence to bond with base pairs on the host DNA. The sequence is not part of the Cas9 protein and as a result is customizable and can be independently synthesized.[15][16]

The PAM sequence on the host genome is recognized by Cas9. Cas9 cannot be easily modified to recognize a different PAM sequence. However this is not too limiting as it is a short sequence and nonspecific (e.g. the SpCas9 PAM sequence is 5'-NGG-3' and in the human genome occurs roughly every 8 to 12 base pairs).[13]

Once these have been assembled into a plasmid and transfected into cells the Cas9 protein with the help of the crRNA finds the correct sequence in the host cell's DNA and depending on the Cas9 variant creates a single or double strand break in the DNA.[17]

Properly spaced single strand breaks in the host DNA can trigger homology directed repair, which is less error prone than the non-homologous end joining that typically follows a double strand break. Providing a DNA repair template allows for the insertion of a specific DNA sequence at an exact location within the genome. The repair template should extend 40 to 90 base pairs beyond the Cas9 induced DNA break.[13] The goal is for the cell's HDR process to utilize the provided repair template and thereby incorporate the new sequence into the genome. Once incorporated, this new sequence is now part of the cell's genetic material and passes into its daughter cells.

Many online tools are available to aid in designing effective sgRNA sequences.[18][19]

Delivery of Cas9, sgRNA, and associated complexes into cells can occur via viral and non-viral systems. Electroporation of DNA, RNA, or ribonucleocomplexes is a common technique, though it can result in harmful effects on the target cells.[20] Chemical transfection techniques utilizing lipids have also been used to introduce sgRNA in complex with Cas9 into cells.[21] Hard-to-transfect cells (e.g. stem cells, neurons, and hematopoietic cells) require more efficient delivery systems such as those based on lentivirus (LVs), adenovirus (AdV) and adeno-associated virus (AAV).[22][23]

Several variants of CRISPR-Cas9 allow gene activation or genome editing with an external trigger such as light or small molecules.[24][25][26] These include photoactivatable CRISPR systems developed by fusing light-responsive protein partners with an activator domain and a dCas9 for gene activation,[27][28] or fusing similar light responsive domains with two constructs of split-Cas9,[29][30] or by incorporating caged unnatural amino acids into Cas9,[31] or by modifying the guide RNAs with photocleavable complements for genome editing.[32]

Methods to control genome editing with small molecules include an allosteric Cas9, with no detectable background editing, that will activate binding and cleavage upon the addition of 4-hydroxytamoxifen (4-HT),[24] 4-HT responsive intein-linked Cas9s[33] or a Cas9 that is 4-HT responsive when fused to four ERT2 domains.[34] Intein-inducible split-Cas9 allows dimerization of Cas9 fragments[35] and Rapamycin-inducible split-Cas9 system developed by fusing two constructs of split Cas9 with FRB and FKBP fragments.[36] Furthermore, other studies have shown to induce transcription of Cas9 with a small molecule, doxycycline.[37][38] Small molecules can also be used to improve Homology Directed Repair (HDR),[39] often by inhibiting the Non-Homologous End Joining (NHEJ) pathway.[40] These systems allow conditional control of CRISPR activity for improved precision, efficiency and spatiotemporal control.

Cas9 genomic modification has allowed for the quick and efficient generation of transgenic models within the field of genetics. Cas9 can be easily introduced into the target cells via plasmid transfection along with sgRNA in order to model the spread of diseases and the cell's response and defense to infection.[41] The ability of Cas9 to be introduced in vivo allows for the creation of more accurate models of gene function, mutation effects, all while avoiding the off-target mutations typically observed with older methods of genetic engineering. The CRISPR and Cas9 revolution in genomic modeling doesn't only extend to mammals. Traditional genomic models such as Drosophila melanogaster, one of the first model species, have seen further refinement in their resolution with the use of Cas9.[41] Cas9 uses cell-specific promoters allowing a controlled use of the Cas9. Cas9 is an accurate method of treating diseases due to the targeting of the Cas9 enzyme only affecting certain cell types. The cells undergoing the Cas9 therapy can also be removed and reintroduced to provide amplified effects of the therapy.[42]

CRISPR-Cas9 can be used to edit the DNA of organisms in vivo and entire chromosomes can be eliminated from an organism at any point in its development. Chromosomes that have been deleted in vivo are the Y chromosomes and X chromosomes of adult lab mice and human chromosomes 14 and 21, in embryonic stem cell lines and aneuploid mice respectively. This method might be useful for treating genetic aneuploid diseases such as Down Syndrome and intersex disorders.[43]

Successful in vivo genome editing using CRISPR-Cas9 has been shown in several model organisms, such as Escherichia coli,[44] Saccharomyces cerevisiae,[45] Candida albicans,[46] Caenorhadbitis elegans,[47] Arabidopsis,[48] Danio rerio,[49] Mus musculus.[50][51] Successes have been achieved in the study of basic biology, in the creation of disease models,[47] and in the experimental treatment of disease models.[52]

Concerns have been raised that off-target effects (editing of genes besides the ones intended) may obscure the results of a CRISPR gene editing experiment (the observed phenotypic change may not be due to modifying the target gene, but some other gene). Modifications to CRISPR have been made to minimize the possibility of off-target effects. In addition, orthogonal CRISPR experiments are recommended to confirm the results of a gene editing experiment.[53][54]

CRISPR simplifies creation of animals for research that mimic disease or show what happens when a gene is knocked down or mutated. CRISPR may be used at the germline level to create animals where the gene is changed everywhere, or it may be targeted at non-germline cells.[55][56][57]

CRISPR can be utilized to create human cellular models of disease. For instance, applied to human pluripotent stem cells CRISPR introduced targeted mutations in genes relevant to polycystic kidney disease (PKD) and focal segmental glomerulosclerosis (FSGS).[58] These CRISPR-modified pluripotent stem cells were subsequently grown into human kidney organoids that exhibited disease-specific phenotypes. Kidney organoids from stem cells with PKD mutations formed large, translucent cyst structures from kidney tubules. The cysts were capable of reaching macroscopic dimensions, up to one centimeter in diameter.[59] Kidney organoids with mutations in a gene linked to FSGS developed junctional defects between podocytes, the filtering cells affected in that disease. This was traced to the inability of podocytes ability to form microvilli between adjacent cells.[60] Importantly, these disease phenotypes were absent in control organoids of identical genetic background, but lacking the CRISPR modifications.[58]

A similar approach was taken to model long QT syndrome in cardiomyocytes derived from pluripotent stem cells.[61] These CRISPR-generated cellular models, with isogenic controls, provide a new way to study human disease and test drugs.

CRISPR-Cas technology has been proposed as a treatment for multiple human diseases, especially those with a genetic cause.[62] Its ability to modify specific DNA sequences makes it a tool with potential to fix disease-causing mutations. Early research in animal models suggest that therapies based on CRISPR technology have potential to treat a wide range of diseases,[63] including cancer,[64] beta-thalassemia,[65] sickle cell disease,[66] hemophilia,[67] cystic fibrosis,[68] Duchenne's muscular dystrophy,[69] Huntington's,[70][71] and heart disease.[72] CRISPR may have applications in tissue engineering and regenerative medicine, such as by creating human blood vessels that lack expression of MHC class II proteins, which often cause transplant rejection.[73]

CRISPR-Cas-based "RNA-guided nucleases" can be used to target virulence factors, genes encoding antibiotic resistance and other medically relevant sequences of interest. This technology thus represents a novel form of antimicrobial therapy and a strategy by which to manipulate bacterial populations.[74][75] Recent studies suggested a correlation between the interfering of the CRISPR-Cas locus and acquisition of antibiotic resistance[76] This system provides protection of bacteria against invading foreign DNA, such as transposons, bacteriophages and plasmids. This system was shown to be a strong selective pressure for the acquisition of antibiotic resistance and virulence factor in bacterial pathogens.[76]

Therapies based on CRISPRCas3 gene editing technology delivered by engineered bacteriophages could be used to destroy targeted DNA in pathogens. [77] Cas3 is more destructive than the better known Cas9[78][79]

Research suggests that CRISPR is an effective way to limit replication of multiple herpesviruses. It was able to eradicate viral DNA in the case of Epstein-Barr virus (EBV). Anti-herpesvirus CRISPRs have promising applications such as removing cancer-causing EBV from tumor cells, helping rid donated organs for immunocompromised patients of viral invaders, or preventing cold sore outbreaks and recurrent eye infections by blocking HSV-1 reactivation. As of August2016[update], these were awaiting testing.[80]

CRISPR may revive the concept of transplanting animal organs into people. Retroviruses present in animal genomes could harm transplant recipients. In 2015, a team eliminated 62 copies of a retrovirus's DNA from the pig genome in a kidney epithelial cell.[81] Researchers recently demonstrated the ability to birth live pig specimens after removing these retroviruses from their genome using CRISPR for the first time.[82]

As of 2016[update] CRISPR had been studied in animal models and cancer cell lines, to learn if it can be used to repair or thwart mutated genes that cause cancer.[83]

The first clinical trial involving CRISPR started in 2016. It involved removing immune cells from people with lung cancer, using CRISPR to edit out the gene expressed PD-1, then administrating the altered cells back to the same person. 20 other trials were under way or nearly ready, mostly in China, as of 2017[update].[64]

In 2016, the United States Food and Drug Administration (FDA) approved a clinical trial in which CRISPR would be used to alter T cells extracted from people with different kinds of cancer and then administer those engineered T cells back to the same people.[84]

Using "dead" versions of Cas9 (dCas9) eliminates CRISPR's DNA-cutting ability, while preserving its ability to target desirable sequences. Multiple groups added various regulatory factors to dCas9s, enabling them to turn almost any gene on or off or adjust its level of activity.[81] Like RNAi, CRISPR interference (CRISPRi) turns off genes in a reversible fashion by targeting, but not cutting a site. The targeted site is methylated, epigenetically modifying the gene. This modification inhibits transcription. These precisely placed modifications may then be used to regulate the effects on gene expressions and DNA dynamics after the inhibition of certain genome sequences within DNA. Within the past few years, epigenetic marks in different human cells have been closely researched and certain patterns within the marks have been found to correlate with everything ranging from tumor growth to brain activity.[6] Conversely, CRISPR-mediated activation (CRISPRa) promotes gene transcription.[85] Cas9 is an effective way of targeting and silencing specific genes at the DNA level.[86] In bacteria, the presence of Cas9 alone is enough to block transcription. For mammalian applications, a section of protein is added. Its guide RNA targets regulatory DNA sequences called promoters that immediately precede the target gene.[87]

Cas9 was used to carry synthetic transcription factors that activated specific human genes. The technique achieved a strong effect by targeting multiple CRISPR constructs to slightly different locations on the gene's promoter.[87]

In 2016, researchers demonstrated that CRISPR from an ordinary mouth bacterium could be used to edit RNA. The researchers searched databases containing hundreds of millions of genetic sequences for those that resembled Crispr genes. They considered the fusobacteria Leptotrichia shahii. It had a group of genes that resembled CRISPR genes, but with important differences. When the researchers equipped other bacteria with these genes, which they called C2c2, they found that the organisms gained a novel defense.[88]

Many viruses encode their genetic information in RNA rather than DNA that they repurpose to make new viruses. HIV and poliovirus are such viruses. Bacteria with C2c2 make molecules that can dismember RNA, destroying the virus. Tailoring these genes opened any RNA molecule to editing.[88]

CRISPR-Cas systems can also be employed for editing of micro-RNA and long-noncoding RNA genes in plants.[89]

Gene drives may provide a powerful tool to restore balance of ecosystems by eliminating invasive species. Concerns regarding efficacy, unintended consequences in the target species as well as non-target species have been raised particularly in the potential for accidental release from laboratories into the wild. Scientists have proposed several safeguards for ensuring the containment of experimental gene drives including molecular, reproductive, and ecological.[90] Many recommend that immunization and reversal drives be developed in tandem with gene drives in order to overwrite their effects if necessary.[91] There remains consensus that long-term effects must be studied more thoroughly particularly in the potential for ecological disruption that cannot be corrected with reversal drives.[92]

Unenriched sequencing libraries often have abundant undesired sequences. Cas9 can specifically deplete the undesired sequences with double strand breakage with up to 99% efficiency and without significant off-target effects as seen with restriction enzymes. Treatment with Cas9 can deplete abundant rRNA while increasing pathogen sensitivity in RNA-seq libraries.[93]

As of November2013[update], SAGE Labs (part of Horizon Discovery group) had exclusive rights from one of those companies to produce and sell genetically engineered rats and non-exclusive rights for mouse and rabbit models.[94] By 2015[update], Thermo Fisher Scientific had licensed intellectual property from ToolGen to develop CRISPR reagent kits.[95]

As of December2014[update], patent rights to CRISPR were contested. Several companies formed to develop related drugs and research tools.[96] As companies ramp up financing, doubts as to whether CRISPR can be quickly monetized were raised.[97] In February 2017 the US Patent Office ruled on a patent interference case brought by University of California with respect to patents issued to the Broad Institute, and found that the Broad patents, with claims covering the application of CRISPR-Cas9 in eukaryotic cells, were distinct from the inventions claimed by University of California.[98][99][100]Shortly after, University of California filed an appeal of this ruling.[101][102]

In March 2017, the European Patent Office (EPO) announced its intention to allow broad claims for editing all kinds of cells to Max-Planck Institute in Berlin, University of California, and University of Vienna,[103][104] and in August 2017, the EPO announced its intention to allow CRISPR claims in a patent application that MilliporeSigma had filed.[103] As of August2017[update] the patent situation in Europe was complex, with MilliporeSigma, ToolGen, Vilnius University, and Harvard contending for claims, along with University of California and Broad.[105]

As of March 2015, multiple groups had announced ongoing research to learn how they one day might apply CRISPR to human embryos, including labs in the US, China, and the UK, as well as US biotechnology company OvaScience.[106] Scientists, including a CRISPR co-discoverer, urged a worldwide moratorium on applying CRISPR to the human germline, especially for clinical use. They said "scientists should avoid even attempting, in lax jurisdictions, germline genome modification for clinical application in humans" until the full implications "are discussed among scientific and governmental organizations".[107][108] These scientists support further low-level research on CRISPR and do not see CRISPR as developed enough for any clinical use in making heritable changes to humans.[109]

In April 2015, Chinese scientists reported results of an attempt to alter the DNA of non-viable human embryos using CRISPR to correct a mutation that causes beta thalassemia, a lethal heritable disorder.[110][111] The study had previously been rejected by both Nature and Science in part because of ethical concerns.[112] The experiments resulted in successfully changing only some of the intended genes, and had off-target effects on other genes. The researchers stated that CRISPR is not ready for clinical application in reproductive medicine.[112] In April 2016, Chinese scientists were reported to have made a second unsuccessful attempt to alter the DNA of non-viable human embryos using CRISPR - this time to alter the CCR5 gene to make the embryo HIV resistant.[113]

In December 2015, an International Summit on Human Gene Editing took place in Washington under the guidance of David Baltimore. Members of national scientific academies of America, Britain and China discussed the ethics of germline modification. They agreed to support basic and clinical research under certain legal and ethical guidelines. A specific distinction was made between somatic cells, where the effects of edits are limited to a single individual, versus germline cells, where genome changes could be inherited by descendants. Heritable modifications could have unintended and far-reaching consequences for human evolution, genetically (e.g. gene/environment interactions) and culturally (e.g. Social Darwinism). Altering of gametocytes and embryos to generate inheritable changes in humans was defined to be irresponsible. The group agreed to initiate an international forum to address such concerns and harmonize regulations across countries.[114]

In November 2018, Jiankui He announced that he had edited two human embryos, to attempt to disable the gene for CCR5, which codes for a receptor that HIV uses to enter cells. He said that twin girls, Lulu and Nana, had been born a few weeks earlier. He said that the girls still carried functional copies of CCR5 along with disabled CCR5 (mosaicism) and were still vulnerable to HIV. The work was widely condemned as unethical, dangerous, and premature.[115] An international group of scientists called for a global moratorium on genetically editing human embryos.[116]

Policy regulations for the CRISPR-Cas9 system vary around the globe. In February 2016, British scientists were given permission by regulators to genetically modify human embryos by using CRISPR-Cas9 and related techniques. However, researchers were forbidden from implanting the embryos and the embryos were to be destroyed after seven days.[117]

The US has an elaborate, interdepartmental regulatory system to evaluate new genetically modified foods and crops. For example, the Agriculture Risk Protection Act of 2000 gives the USDA the authority to oversee the detection, control, eradication, suppression, prevention, or retardation of the spread of plant pests or noxious weeds to protect the agriculture, environment and economy of the US. The act regulates any genetically modified organism that utilizes the genome of a predefined "plant pest" or any plant not previously categorized.[118] In 2015, Yinong Yang successfully deactivated 16 specific genes in the white button mushroom, to make them non-browning. Since he had not added any foreign-species (transgenic) DNA to his organism, the mushroom could not be regulated by the USDA under Section 340.2.[119] Yang's white button mushroom was the first organism genetically modified with the CRISPR-Cas9 protein system to pass US regulation.[120] In 2016, the USDA sponsored a committee to consider future regulatory policy for upcoming genetic modification techniques. With the help of the US National Academies of Sciences, Engineering and Medicine, special interests groups met on April 15 to contemplate the possible advancements in genetic engineering within the next five years and any new regulations that might be needed as a result.[121] The FDA in 2017 proposed a rule that would classify genetic engineering modifications to animals as "animal drugs", subjecting them to strict regulation if offered for sale, and reducing the ability for individuals and small businesses to make them profitably.[122][123]

In China, where social conditions sharply contrast with the west, genetic diseases carry a heavy stigma.[124] This leaves China with fewer policy barriers to the use of this technology.[125][126]

In 2012, and 2013, CRISPR was a runner-up in Science Magazine's Breakthrough of the Year award. In 2015, it was the winner of that award.[81] CRISPR was named as one of MIT Technology Review's 10 breakthrough technologies in 2014 and 2016.[127][128] In 2016, Jennifer Doudna, Emmanuelle Charpentier, along with Rudolph Barrangou, Philippe Horvath, and Feng Zhang won the Gairdner International award. In 2017, Jennifer Doudna and Emmanuelle Charpentier were awarded the Japan Prize for their revolutionary invention of CRISPR-Cas9 in Tokyo, Japan. In 2016, Emmanuelle Charpentier, Jennifer Doudna, and Feng Zhang won the Tang Prize in Biopharmaceutical Science.[129]

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Jennifer Doudna: We will eat the first Crispr’d food In 5 …

While ethicists debate the applications of blockbuster gene-editing tool Crispr in human healthcare, an inventor of the tool believes it has a more immediate application: improving our food.

"I think in the next five years the most profound thing we'll see in terms of Crispr's effects on people's everyday lives will be in the agricultural sector," Jennifer Doudna, the University of California Berkeley geneticist who unearthed Crispr in early experiments with bacteria in 2012, told Business Insider.

Crispr has dozens of potential uses, from treating diseases like sickle cell to certain inherited forms of blindness. The tool recently made headlines when a scientist in China reportedly used it to edit the DNA of a pair of twin baby girls.

Then there are Crispr's practical applications the kinds of things we might expect to see in places like grocery stores and farmers' fields within a decade, according to Doudna.

Crispr's appeal in food is straightforward: it's cheaper and easier than traditional breeding methods, including those that are used to make genetically modified crops (also known as GMOs) currently. It's also much more precise. Where traditional breeding methods hack away at a crop's genome with a dull blade, tools like Crispr slice and reshape with scalpel-like precision.

Want a mushroom that doesn't brown? A corn crop that yields more food per acre? Both already exist, though they haven't yet made it to consumers' plates. What about a strawberry with a longer shelf life or tomatoes that do a better job of staying on the vine?

"I think all of those things are coming relatively quickly," Doudna said.

Read more: The 10 people transforming healthcare

Work on Crispr'd produce has been ongoing for about half a decade, but it's only recently that US regulators have created a viable path for Crispr'd products to come to market.

Back in 2016, researchers at Penn State used Crispr to make mushrooms that don't brown. Last spring, gene-editing startup Pairwise scored $125 million from agricultural giant Monsanto to work on Crispr'd produce with the goal of getting it in grocery stores within the decade. A month later, Stefan Jansson, the chief of the plant physiology department at Sweden's Umea University, grew and ate the world's first Crispr'd kale.

More recently, several Silicon Valley startups have been experimenting with using Crispr to make lab-grown meat.

Read more: Startups backed by celebrities like Bill Gates are using Crispr to make meat without farms

Memphis Meats, a startup with backing from notable figures like Bill Gates and Richard Branson that has made real chicken strips and meatball prototypes from animal cells (and without killing any animals), is using the tool. So is New Age Meats, another San Francisco-based startup that aims to create real meat without slaughter.

Last spring, the US Department of Agriculture issued a new ruling on crops that exempts many Crispr-modified crops from the oversight that accompanies traditional GMOs. So long as the edited DNA in those crops could also have been created using traditional breeding techniques, the Crispr'd goods are not subject to those additional regulatory steps, according to the agency.

"With this approach, USDA seeks to allow innovation when there is no risk present," secretary of agriculture Sonny Perdue said in a statement. Genome editing tools like Crispr, he added, "will help farmers do what we aspire to do at USDA: do right and feed everyone."

Read more: A controversial technology could save us from starvation if we let it

Although several researchers and scientists have cheered the decision, many anti-GMO activists have not been pleased.

Despite the pushback, Doudna believes that Crispr'd food could help dispel some of the fear around GMOs and increase awareness about the role of science in agriculture.

"I hope this brings that discussion into a realm where we can talk about it in a logical way," she said. "Isn't it better to have technology that allows for precise manipulation of a plant genome, rather than relying on random changes?"

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Cell Therapy World Asia 2019 – IMAPAC – Imagine your impact

Cell Therapy World Asia 2019

Asia-Pacifics ONLY Cell Therapy Focused Regional Event!

Tokyo, Japan

Cell Therapy World Asia 2019 is bringing together Asias best of best in cell therapy development and manufacturing. This will be the most targeted and the only regional conference that will attract cell therapy companies in South Korea, Japan, China, India, Singapore, Taiwan and the rest of Asia to discuss and debate on best practices and innovations in this space.

Event Highlights200+Key Stakeholders from TOP Cell Therapy Companies 50+ Asia-Pacificcell therapy companies to attend 30+ Key opinion leaders to share their insights 20+ Hours of Networking 15+ Technology Showcase

What is in it for you?

Sales and Marketing Opportunities @ Cell Therapy WorldAsia 2019

To ensure your target audience in Korea and Asia gets to hear your product philosophy and successful case studies at the conference, its important to discuss with us about your potential involvement early! Get involved by taking your first step, contact:

Speaking OpportunitiesAarthi AsokanConference ProducerT: (65) 3109 0159E: aarthi.asokan@imapac.com

Sponsorship OpportunitiesMatthew YongBusiness Development ManagerT: (65) 3109 0123E: matthew.yong@imapac.com

Delegate & Media RegistrationAkanksha MittalMarketing ManagerT: (65) 3109 0158E: akanksha.mittal@imapac.com

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Gene therapy restores immunity in infants with rare …

News Release

Wednesday, April 17, 2019

NIH scientists and funding contributed to development of experimental treatment

A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune cells do not develop or function normally. Eight infants with the disorder, called X-linked severe combined immunodeficiency (X-SCID), received an experimental gene therapy co-developed by National Institutes of Health scientists. They experienced substantial improvements in immune system function and were growing normally up to two years after treatment. The new approach appears safer and more effective than previously tested gene-therapy strategies for X-SCID.

These interim results from the clinical trial, supported in part by NIH, were published today in The New England Journal of Medicine.

Infants with X-SCID, caused by mutations in the IL2RG gene, are highly susceptible to severe infections. If untreated, the disease is fatal, usually within the first year or two of life. Infants with X-SCID typically are treated with transplants of blood-forming stem cells, ideally from a genetically matched sibling. However, less than 20% of infants with the disease have such a donor. Those without a matched sibling typically receive transplants from a parent or other donor, which are lifesaving, but often only partially restore immunity. These patients require lifelong treatment and may continue to experience complex medical problems, including chronic infections.

"A diagnosis of X-linked severe combined immunodeficiency can be traumatic for families," said Anthony S. Fauci, M.D., director of NIHs National Institute of Allergy and Infectious Diseases (NIAID). These exciting new results suggest that gene therapy may be an effective treatment option for infants with this extremely serious condition, particularly those who lack an optimal donor for stem cell transplant. This advance offers them the hope of developing a wholly functional immune system and the chance to live a full, healthy life.

To restore immune function to those with X-SCID, scientists at NIAID and St. Jude Childrens Research Hospital in Memphis, Tennessee, developed an experimental gene therapy that involves inserting a normal copy of the IL2RG gene into the patients own blood-forming stem cells. The Phase 1/2 trial reported today enrolled eight infants aged 2 to 14 months who were newly diagnosed with X-SCID and lacked a genetically matched sibling donor. The study was conducted at St. Jude and the Benioff Childrens Hospital of the University of California, San Francisco. Encouraging early results from a separate NIAID-led study at the NIH Clinical Center informed the design of the study in infants. The NIH study is evaluating the gene therapy in older children and young adults with X-SCID who previously had received stem cell transplants.

The gene therapy approach involves first obtaining blood-forming stem cells from a patients bone marrow. Then, an engineered lentivirus that cannot cause illness is used as a carrier, or vector, to deliver the normal IL2RGgene to the cells. Finally, the stem cells are infused back into the patient, who has received a low dose of the chemotherapy medication busulfan to help the genetically corrected stem cells establish themselves in the bone marrow and begin producing new blood cells.

Normal numbers of multiple types of immune cells, including T cells, B cells and natural killer (NK) cells, developed within three to four months after gene therapy in seven of the eight infants. While the eighth participant initially had low numbers of T cells, the numbers greatly increased following a second infusion of the genetically modified stem cells. Viral and bacterial infections that participants had prior to treatment resolved afterwards. The experimental gene therapy was safe overall, according to the researchers, although some participants experienced expected side effects such as a low platelet count following chemotherapy.

"The broad scope of immune function that our gene therapy approach has restored to infants with X-SCID as well as to older children and young adults in our study at NIH is unprecedented," said Harry Malech, M.D., chief of the Genetic Immunotherapy Section in NIAIDs Laboratory of Clinical Immunology and Microbiology. Dr. Malech co-led the development of the lentiviral gene therapy approach with St. Judes Brian Sorrentino, M.D., who died in late 2018. These encouraging results would not have been possible without the efforts of my good friend and collaborator, the late Brian Sorrentino, who was instrumental in developing this treatment and bringing it into clinical trials, said Dr. Malech.

Compared with previously tested gene-therapy strategies for X-SCID, which used other vectors and chemotherapy regimens, the current approach appears safer and more effective. In these earlier studies, gene therapy restored T cell function but did not fully restore the function of other key immune cells, including B cells and NK cells. In the current study, not only did participants develop NK cells and B cells, but four infants were able to discontinue treatment with intravenous immunoglobulins infusions of antibodies to boost immunity. Three of the four developed antibody responses to childhood vaccinations an indication of robust B-cell function.

Moreover, some participants in certain early gene therapy studies later developed leukemia, which scientists suspect was because the vector activated genes that control cell growth. The lentiviral vector used in the study reported today is designed to avoid this outcome.

Researchers are continuing to monitor the infants who received the lentiviral gene therapy to evaluate the durability of immune reconstitution and assess potential long-term side effects of the treatment. They also are enrolling additional infants into the trial. The companion NIH trial evaluating the gene therapy in older children and young adults also is continuing to enroll participants.

The gene therapy trial in infants is funded by the American Lebanese Syrian Associated Charities (ALSAC), and by grants from the California Institute of Regenerative Medicine and the National Heart, Lung, and Blood Institute, part of NIH, under award number HL053749. The work also is supported by NIAID under award numbers AI00988 and AI082973, and by the Assisi Foundation of Memphis. More information about the trial in infants is available on ClinicalTrials.gov using identifier NCT01512888. More information about the companion trial evaluating the treatment in older children and young adults is available using ClinicalTrials.gov identifier NCT01306019.

NIAID conducts and supports research at NIH, throughout the United States, and worldwide to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on the NIAID website.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

E Mamcarz et al. Lentiviral gene therapy with low dose busulfan for infants with X-SCID. The New England Journal of Medicine DOI: 10.1056/NEJMoa1815408 (2019).

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Bubble boy disease: Doctors successfully treat SCID-X1 …

Researchers from St. Jude Childrens Research Hospital have cured babies with bubble boy disease through gene therapy. Angela Gosnell, Knoxville News Sentinel

MEMPHIS, Tenn. Researchers from St. Jude Childrens Research Hospital have cured babies with bubble boy disease through gene therapy involving a re-engineeredvirus, according to a newly published study.

St. Jude performed the therapy oninfants newly diagnosed withX-linked severe combined immunodeficiency (SCID-X1) a genetic condition also known as "bubble boy" disease according to a study published in the New England Journal of Medicine's April 18 issue.

The diseaseprevents babies from developing an immune system to fight even routine infections.In January 2018, St. Jude researchers reported that babies in the trial developed fully functioning immune systems but would be monitored further to confirm its long-term benefits.

Corresponding authors Dr. Ewelina Mamcarz and Dr. Stephen Gottschalk from St. Jude Children's Research Hospital. St. Jude performed a new therapy oninfants newly diagnosed withX-linked severe combined immunodeficiency (SCID-X1), a genetic condition called "bubble boy" disease, according to a study published in the New England Journal of Medicine's April 18 issue.(Photo: Peter Barta / St. Jude Childrens Research Hospital)

Previous infections cleared in all infants, and all continued to grow normally, the study said of the results.

St. Jude and UCSF Benioff Childrens Hospital San Francisco treated the children enrolled in the clinical trial with gene therapy developed by St. Judes Brian Sorrentino, the studys senior author,who led groundbreaking gene therapy research before his death in November at 60 years old.

Brian Sorrentino(Photo: Courtesy of Memorial Park Funeral Home)

James Downing, CEO of St. Jude Children's Research Hospital, said it was the lifelong ambition of Sorrentino, a survivor of pediatric cancer, to develop a cure.

Were comfortable, I think, at this point stating this is a cure, Downing said. Only time will say this will be a durable, lifelong cure.

After the therapy, the babies received their standard vaccinations and are now living a normal life with fully functioning immune systems, St. Jude says. Ten infants have received the therapy so far.

Study co-author Stephen Gottschalk, chair of the St. Jude Department of Bone Marrow Transplantation and Cellular Therapy, said the researchers hope the therapy will be a template for treating other blood disorders.

Newborns with bubble boy disease, caused by a mutation inside a specific gene,must be placed inprotective isolation because they lack a proper immune system. Contact with the outside world is a major infection risk.

Perhaps the most well-known person with the disease was David Vetter, who died in 1984 at 12 years old. He helped inspire the 1976 movie "The Boy in the Plastic Bubble."

David Vetter had to stay inside a bubble in Houston on Dec. 17, 1976. Vetter was born with a genetic disorder leaving him no natural immunity against disease. Vetter died in 1984.(Photo: AP)

Most with the disease die by age 2 without treatment.

This disease is called bubble boy disease because babies had to be kept in special plastic chambers to protect them from infections, said first and corresponding author Ewelina Mamcarzof the St. Jude Department of Bone Marrow Transplantation and Cellular Therapy. We dont have these chambers now, we are more advanced, but we need to protect them from infections as simple as a common cold virus (that) can kill them.

The patients came to researchers between 2 and 14 months of age, Mamcarz said, with severe life-threatening infections.

The gene therapy works like this: A deactivated virus is inserted into the patients bone marrow, which deliversthe correct gene copy into blood stem cells, replacing the defective one. These cells are then frozen and undergo testing.

This virus is able to effectively deliver a healthy copy of the gene into a stem cell in a way that was not possible before, Mamcarz said.

The patient then receives two days of low-dose busulfan, a chemotherapy drug that makes space in the marrow for the stem cells to grow, and the cells are then infused back into the patient.

Dr. Ewelina Mamcarz, first and corresponding author of a study published in the New England Journal of Medicine about a therapy performed at St. Jude Children's Research Hospital oninfants newly diagnosed withX-linked severe combined immunodeficiency (SCID-X1), a genetic condition called "bubble boy" disease.(Photo: Peter Barta / St. Jude Childrens Research Hospital)

It takes about 10 days from the time the cells are taken outto when they are infused into the patient, Mamcarz said.

The proper immune cells were found within three months of the treatment in all but one patient, who needed a second dose of gene therapy, St. Jude says.

This novel approach has shown really outstanding results for the infants, Downing said. The treatment has fully restored the immune system in these patients, which wasnt possible before, and has no immediate side effects.

The gene therapy developed and produced at St. Jude differs from previous gene replacement efforts in part by not activating adjacent genes that could cause leukemia. The viruses are equipped with insulators to block that accidental activation.

Past gene therapy did not have insulators, which inadvertently caused leukemia, Gottschalk said.

Gael Jesus Pino Alva, 2, and his mother, Giannina Alva. Gael was treated with a new therapy designed to fight X-linked severe combined immunodeficiency (SCID-X1), a genetic condition known as "bubble boy" disease, at St. Jude Children's Research Hospital.(Photo: Peter Barta / St. Jude Childrens Research Hospital)

Current treatments for bubble boydisease are limited. Bone marrow transplants from compatible sibling donors are the best bet, but most patientslack a properdonor.

Mamcarz said researchers would like to treat more patients and follow them for longer periods of time to see if the gene therapy performed in the clinicaltrial can truly be used as an upfront treatment, and it's still too early to determine costs.

But the results from the research are a first, and their approach could be used to eventually treatother disorders like sickle cell disease, she said.

The kids are cured because for the first time, we are able to restore all three types of cells that constitute a full immune system: T cells, B cells and NK cells, Mamcarz said. Our patients are able to generate a healthy, fully functioning immune system. That is the first for gene therapy.

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Downingsaid the search for a cure has been a journey spanning more than a decade. Early gene therapy studies with the viral vectorsled to leukemia, he said, causing the work to stall. But Sorrentino pushed on.

Brian Sorrentino decided we really needed to produce vectors we could trust in not inducing leukemia, Downingsaid.

The patients' quality of life following the treatmentshows theyindeed found a cure, Downing said.

The question will become, Will it be a durable cure? Will it last 10, 20, 50 years for these children? And only time will tell," he said.

Follow Max Garland on Twitter:@MaxGarlandTypes.

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Crispr Gene Editing Is Coming for the Womb | WIRED

William Peranteau is the guy parents call when theyve received the kind of bad news that sinks stomachs and wrenches hearts. Sometimes its a shadow on an ultrasound or a few base pairs out of place on a prenatal genetic test, revealing that an unborn child has a life-threatening developmental defect. Pediatric surgeons like Peranteau, who works at Childrens Hospital of Philadelphia, usually cant try to fix these abnormalities until their patients leave their mothers bodies behind. And by then it might be too late.

Its with the memory of the families he couldnt help in the back of his mind that Peranteau has joined a small group of scientists trying to bring the fast-moving field of gene editing to the womb. Such editing in humans is a long way off, but a spate of recent advances in mouse studies highlight its potential advantages over other methods of using Crispr to snip away diseases. Parents confronted with an in utero diagnosis are often faced with only two options: terminate the pregnancy or prepare to care for a child who may require multiple invasive surgeries over the course of their lifetime just to survive. Prenatal gene editing may offer a third potential path. What we see as the future is a minimally invasive way of treating these abnormalities at their genetic origin instead, says Peranteau.

To prove out this vision, Peranteau and colleagues at the University of Pennsylvania injected Crispr editing components, encoded in a virus, into the placentas of pregnant mice whose unborn pups were afflicted with a lethal lung-disease-causing mutation. When the fetuses breathed in the amniotic fluid they also inhaled the Crispr bits, which went to work editing the DNA inside their rapidly dividing alveolar progenitor cells. These cells give rise to many types of cells that line the lungsincluding ones that secrete a sticky substance that keeps the lungs from collapsing every time you breathe. Mutations to proteins that make up this secretion are a major source of congenital respiratory conditions. All of the mice with the mutation died within a few hours of birth. Of those edited with Crispr, about a quarter survived. The results were published in todays issue of Science Translational Medicine.

Its the second proof of concept from the group of scientists in the past year. In October, they published a paper describing a slightly different procedure to edit mutations that lead to a lethal metabolic disorder. By changing a single base pair in the liver cells of prenatal mice, Peranteaus team was able to rescue nearly all of the mouse pups. Other recent successes include unborn mice cured of a blood disorder called beta-thalassemia following a prenatal injection of Crispr, carried out last year by a team at Yale and Carnegie Mellon.

Though the field is still in its infancy, its pioneers believe that many of the problems Crispr-based therapies have to contend withlike reaching enough of the right cells and evading the human immune systemcan be solved by treating patients while they are still in the womb.

If youre trying to edit cells in an adult organ, theyre not proliferating, so you have to reach a lot of them to have any impact, says Edward Morrisey, a cardiologist at the University of Pennsylvania, who coauthored the latest study. Fetuses, on the other hand, are still developing, which means their cells are in a state of rapid division as they grow into new tissues. The earlier in life you can edit, the more those genetic changes will multiply and propagate through developing organs. Morriseys mice might have only been born with the genetic edit in about 20 percent of their lung cells, but 13 weeks later, the correction had spread to the entire surface of the lung. Theyve actually outcompeted the nonedited cells, because those cells are very sick, says Morissey.

For lung diseases in particular, this represents a huge advantage. As soon as a baby leaves the watery world of the womb, its lung cells start secreting a barrier of mucus mixed with surfactant, to keep any dust or viruses or other foreign objects, including Crispr components, from reaching those tissues. A developing fetus also has a less aggressive immune system than a human whos been exposed to the outside world. So its less likely to mount an attack on Crispr components, which do, after all, originate in the bacterial kingdom.

Now, you might be thinking, if editing earlier is better, why not edit an embryo right after its been fertilized, when its only a cell or two old? But this technique, known as germline editing (you might remember it from last years Chinese Crispr baby scandal), is a much more complicated ethical endeavor. Editing at that stage would pass on any changes to every cell, including the ones that would go on to make sperm or eggs. This kind of editing is effectively banned in the United States, following a directive from Congress to the US Food and Drug Administration to not allow any clinical trials involving genetically modified human embryos. (The ban, which has to be renewed annually, was most recently reaffirmed in February of 2019). The other thing though, is that getting an accurate diagnosis when an embryo is only a few cells old can be tricky. Waiting long enough to get a visual on a fetus along with other vital signs can provide important clues as to the severity of the condition. It gets us right in that sweet spot to treat a disease at the very beginning, basically as soon as its diagnosed, says Peranteau.

But there are still safety issues to resolve. For one thing, in utero editing involves two patients, not just one. In the process of curing a child, this technique would potentially expose a healthy bystanderthe motherto a treatment that provides no potential benefit and only potential risks, including dangerous immune reactions. And because the editing is taking place inside her reproductive tract, some wayward Crispr components might wend their way up her fallopian tubes and into her ovaries, potentially making changes to other, unfertilized eggs. A lot more science will need to be done to better assess these risks. To give you an idea of how long these things can take, consider that in utero gene therapyan older approach that entails replacing a defective gene with a functioning one using a viruswas first proposed back in the mid-1990s following a series of positive proof of concept studies in mice. Today, only a single clinical trial is in progress.

This is not a panacea for curing every genetic disease thats out there, says Peranteau. But he believes that a Crispr approach will be able to piggyback on the work of the gene therapy field, and may offer a new way forward for at least some of his patients. At some point in the futurenot tomorrow or the next day, years from nowI think in utero editing would provide hope for families that today have none.

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CRISPR Research Moves Out Of Labs And Into Clinics Around The …

CRISPR gene-editing technology allows scientists to make highly precise modifications to DNA. The technology is now starting to be used in human trials to treat several diseases in the U.S. Molekuul/Getty Images/Science Photo Library hide caption

CRISPR gene-editing technology allows scientists to make highly precise modifications to DNA. The technology is now starting to be used in human trials to treat several diseases in the U.S.

The powerful gene-editing technique called CRISPR has been in the news a lot. And not all the news has been good: A Chinese scientist stunned the world last year when he announced he had used CRISPR to create genetically modified babies.

But scientists have long hoped CRISPR a technology that allows scientists to make very precise modifications to DNA could eventually help cure many diseases. And now scientists are taking tangible first steps to make that dream a reality.

For example, NPR has learned that a U.S. CRISPR study that had been approved for cancer at the University of Pennsylvania in Philadelphia has finally started. A university spokesman on Monday confirmed for the first time that two patients had been treated using CRISPR.

One patient had multiple myeloma, and one had sarcoma. Both had relapsed after undergoing standard treatment.

The revelation comes as several other human trials of CRISPR are starting or are set to start in the U.S., Canada and Europe to test CRISPR's efficacy in treating various diseases.

"2019 is the year when the training wheels come off and the world gets to see what CRISPR can really do for the world in the most positive sense," says Fyodor Urnov, a gene-editing scientist at the Altius Institute for Biomedical Sciences in Seattle and the University of California, Berkeley.

Here are highlights of the year ahead in CRISPR research, and answers to common questions about the technology.

What is CRISPR exactly?

CRISPR is a new kind of genetic engineering that gives scientists the power to edit DNA much more easily than ever. Researchers think CRISPR could revolutionize how they prevent and treat many diseases. CRISPR could, for example, enable scientists to repair genetic defects or use genetically modified human cells as therapies.

Traditional gene therapy uses viruses to insert new genes into cells to try to treat diseases. CRISPR treatments largely avoid the use of viruses, which have caused some safety problems in the past. Instead they directly make changes in the DNA, using targeted molecular tools. The technique has been compared to the cut and paste function in a word processing program it allows scientists to remove or modify specific genes causing a problem.

Is this the same technique that caused a recent scandal when a scientist in China edited the genes of two human embryos?

There's an important difference between the medical studies under discussion here and what the Chinese scientist, He Jiankui, did. He used CRISPR to edit genes in human embryos. That means the changes he made would be passed down for generations to come. And he did it before most scientists think it was safe to try. In fact, there have been calls for a moratorium on gene-editing of heritable traits.

For medical treatments, modifications are only being made in the DNA of individual patients. So this gene-editing doesn't raise dystopian fears about re-engineering the human race. And there's been a lot of careful preparation for these studies to avoid unintended consequences.

So what's happening now with new or planned trials?

We've finally reached the moment when CRISPR is moving out of the lab and into the clinic around the world.

Until now, only a relatively small number of studies have tried to use CRISPR to treat disease. And almost all of those studies have been in China, and have been aimed at treating various forms of cancer.

There's now a clinical trial underway at the University of Pennsylvania using CRISPR for cancer treatment. It involves removing immune system cells from patients, genetically modifying them in the lab and infusing the modified cells back into the body.

The hope is the modified cells will target and destroy cancer cells. No other information has been released about how well it might be working. The study was approved to eventually treat 18 patients.

"Findings from this research study will be shared at an appropriate time via medical meeting presentation or peer-reviewed publication," a university spokesperson wrote in an email to NPR.

But beyond the cancer study, researchers in Europe, the United States and Canada are launching at least half a dozen carefully designed studies aimed at using CRISPR to treat a variety of diseases.

What other diseases are they testing treatments for?Two trials sponsored by CRISPR Therapeutics of Cambridge, Mass., and Vertex Pharmaceuticals of Boston are designed to treat genetic blood disorders. One is for sickle cell disease, and another is a similar genetic condition called beta thalassemia.

In fact, the first beta thalassemia patient was recently treated in Germany. More patients may soon get their blood cells edited using CRISPR at that hospital and a second clinic in Germany, followed by patients at medical centers in Toronto, London and possibly elsewhere.

The first sickle disease patients could soon start getting the DNA in their blood cells edited in this country in Nashville, Tenn., San Antonio and New York.

And yet another study, sponsored by Editas Medicine of Cambridge, Mass., will try to treat an inherited form of blindness known as Leber congenital amaurosis.

That study is noteworthy because it would be the first time scientists try using CRISPR to edit genes while they are inside the human body. The other studies involve removing cells from patients, editing the DNA in those cells in the lab and then infusing the modified cells back into patients' bodies.

Finally, several more U.S. cancer studies may also start this year in Texas, New York and elsewhere to try to treat tumors by genetically modifying immune system cells.

What can go wrong with CRISPR? Are there any concerns?

Whenever scientists try something new and powerful, it always raises fears that something could go wrong. The early days of gene therapy were scarred by major setbacks, such as the case of Jesse Gelsinger, who died after an adverse reaction to a treatment.

The big concern about CRISPR is that the editing could go awry, causing unintended changes in DNA that could cause health problems.

There's also some concern about this new wave of studies because they are the first to get approved without going through an extra layer of scrutiny by the National Institutes of Health. That occurred because the NIH and FDA changed their policy, saying only some studies would require that extra layer of review.

"Every human on the planet should hope that this technology works. But it might work. It might not. It's unknown," says Laurie Zoloth, a bioethicist at the University of Chicago. "This is an experiment. So you do need exquisite layers of care. And you need to really think in advance with a careful ethical review how you do this sort of work."

The researchers conducting the studies say they have conducted careful preliminary research, and their studies have gone through extensive scientific and ethical review.

When might we know whether any of these experimental CRISPR treatments are working?

All of these studies are very preliminary and are primarily aimed at first testing whether this is safe. That said, they are also looking for clues to whether they might be helping patients. So there could be at least a hint about that later this year. But it will be many years before any CRISPR treatment could become widely available.

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CRISPR Research Moves Out Of Labs And Into Clinics Around The ...

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CRISPR gene editing has been used on humans in the US

It's not certain how effective the treatment has been, and you won't find out for a while when the trial has been cleared to treat a total of 18 patients. You won't hear more about it until there's been a presentation or a peer-reviewed paper, the university said. Other trials, such as ones for blood disorders in the Boston area, have yet to get underway.

No matter what, any practical uses could take a long time. There are widespread concerns that CRISPR editing could have unanticipated effects, and scientists have yet to try editing cells while they're still in the body (a blindness trial in Cambridge, MA may be the first instance). There's also the not-so-small matter of ethical questions. Chinese scientist He Jiankui raised alarm bells when he said he edited genes in human embryos -- politicians and the scientific community will likely want to address practices like that before you can simply assume that CRISPR is an option.

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CRISPR gene editing has been used on humans in the US

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Gene Therapy 2019 Global Market Outlook,Research,Trends …

WiseGuyReports.Com Publish a New Market Research Report On Gene Therapy 2019 Global Market Outlook,Research,Trends and Forecast to 2026.

Pune, India April 15, 2019

Gene Therapy Industry 2019

Description:-

The global gene therapy market is anticipated to reach USD 4,300 million by 2021. The demand for gene therapy is primarily driven by continuous technological advancements and successful progression of several clinical trials targeting treatments with strong unmet need. Moreover, rising R&D spend on platform technologies by large and emerging biopharmaceutical companies and favorable regulatory environment will accelerate the clinical development and the commercial approval of gene therapies in the foreseeable future. Despite promise, the high cost of gene therapy represents a significant challenge for commercial adoption in the forecast period.

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Gene therapy involves inactivating a mutated gene that is not functioning properly and introducing a new gene to assist in fighting a disease. Overall, the field of gene therapy continues to mature and advance with many products in development and nearing commercialization. For instance, Spark Therapeutics received approval of Luxturna, a rare form inherited blindness in December 2017. Gene therapy market in late 2017 also witnessed the approvals of Gilead/Kite Pharmas Yescarta and Novartis Kymriah in the cancer therapeutic area.

Gene therapy offers promise in the treatment of range of indications in cancer and genetic disorders. Large Pharmaceuticals and Biotechnology companies exhibit strong interest in this field and key among them include Allergan, Shire, Biomarin, Pfizer and GSK. The gene therapy space is witnessing a wave of partnerships and alliances. Pfizer has recently expanded its presence in gene therapy with the acquisition of Bamboo Therapeutics and Allergan entered the field, with the acquisition of RetroSense and its Phase I/II optogenetic program.

North America holds a dominating position in the global gene therapy market which is followed by Europe and the Asia Pacific. The U.S. has maximum number of clinical trials ongoing followed by Europe. Moreover, the field of gene therapy in the U.S. and Europe continues to gain investor attention driven by success of high visible clinical programs and the potential of gene therapy to address strong unmet need with meaningful commercial opportunity. Moreover, the increasing partnerships and alliances and the disruptive potential of gene therapy bodes well for the sector through the forecast period.Key Findings from the study suggest products accessible in the market are much competitive and manufacturers are progressively concentrating on advancements to pick up an aggressive edge. Companies are in a stage of development of new items in order to guarantee simple implementation and connection with the current gene. The hospatility segment is anticipated to grow at a high growth rate over the forecast period with the expanding utilization of smart locks inferable from expanding security-related worries among clients amid their stay at the hotels. North America is presumed to dominate the global smart locks market over the forecast years and Asia Pacific region shows signs of high growth owing to the booming economies of India, and China.

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Table Of Contents Major Key Points

1. Gene Therapy Overview1.1. History and Evolution of Gene Therapies1.2. What is Gene Therapy1.3. Types of Gene Therapy1.4. Ex vivo and in vivo Approaches of Gene Therapy1.5. RNAi Therapeutics1.6. CAR-T Technology based Gene Therapy1.7. Types of Vectors used for Gene Therapy1.7.1. Viral1.7.2. Non-Viral

2. Historical Marketed Gene Therapies [2003-2012]2.1. Rexin-G (Epeius Biotechnologies Corporation)2.2. Gendicine (SiBiono GeneTech Co., Ltd)2.3. Neovasculgen [Human Stem Cells Institute (HSCI))2.4. Glybera (UniQure Biopharma B.V.)

3. First Countries to get an access to Gene Therapies3.1. Philippines for Rexin-G [2003]3.2. China for Gendicine [2003]3.3. Russia for Neovasculgen [2011]3.4. Selected European Countries for Glybera [2012]

4. Marketed Gene Therapies [Approved in Recent Years]4.1. KYMRIAH (tisagenlecleucel)4.1.1. Therapy Description4.1.2. Therapy Profile4.1.2.1. Company4.1.2.2. Approval Date4.1.2.3. Mechanism of Action4.1.2.4. Researched Indication4.1.2.5. Vector Used4.1.2.6. Vector Type4.1.2.7. Technology4.1.2.8. Others Development Activities4.1.3. KYMRIAH Revenue Forecasted till 20214.2. YESCARTA (axicabtagene ciloleucel)4.2.1. Therapy Description4.2.2. Therapy Profile4.2.2.1. Company4.2.2.2. Approval Date4.2.2.3. Mechanism of Action4.2.2.4. Researched Indication4.2.2.5. Vector Used4.2.2.6. Vector Type4.2.2.7. Technology4.2.2.8. Others Development Activities4.2.3. YESCARTA Revenue Forecasted till 20214.3. LUXTURNA (voretigene neparvovec-rzyl)4.3.1. Therapy Description4.3.2. Therapy Profile4.3.2.1. Company4.3.2.2. Approval Date4.3.2.3. Mechanism of Action4.3.2.4. Researched Indication4.3.2.5. Vector Used4.3.2.6. Vector Type4.3.2.7. Technology4.3.2.8. Others Development Activities4.3.3. LUXTURNA Revenue Forecasted till 20214.4. STRIMVELIS4.4.1. Therapy Description4.4.2. Therapy Profile4.4.2.1. Company4.4.2.2. Approval Date4.4.2.3. Mechanism of Action4.4.2.4. Researched Indication4.4.2.5. Vector Used4.4.2.6. Vector Type4.4.2.7. Technology4.4.2.8. Others Development Activities4.4.3. STRIMVELIS Revenue Forecasted till 2021

5. Comparison of current Regulatory Status for Gene Therapy Products5.1. U.S5.2. Europe5.3. Japan

6. Emerging Gene Therapies [Phase III]6.1. Gene Based Therapeutics under Development6.2. Therapy Description

7. Indication of Focus in Gene Therapy7.1. Cancer7.2. Neurodegenerative Disorders7.3. Lysosomal Storage Disorders (LSDs)7.4. Ocular Diseases7.5. Muscle Disorders7.6. Anemia7.7. Hemophilia7.8. Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency

Continued

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Gene Therapy 2019 Global Market Outlook,Research,Trends ...

Recommendation and review posted by Bethany Smith

Crispr Can Speed Up Natureand Change How We Grow Food | WIRED

Like any self-respecting farmer, Zachary Lippman was grumbling about the weather. Stout, with close-cropped hair and beard, Lippman was standing in a greenhouse in the middle of Long Island, surrounded by a profusion of rambunctiously bushy plants. Dont get me started, he said, referring to the late and inclement spring. It was a Tuesday in mid-April, but a chance of snow had been in the forecast, and a chilly wind blew across the island. Not the sort of weather that conjures thoughts of summer tomatoes. But Lippman was thinking ahead to sometime around Memorial Day, when thousands of carefully nurtured tomato plants would make the move from the greenhouse to Long Island loam. He hoped the weather would finally turn.

Although he worked on a farm as a teenager and has a romantic attachment to the soil, Lippman isnt a farmer. Hes a plant biologist at Cold Spring Harbor Laboratory in New York with an expertise in genetics and development. And these greenhouse plants arent ordinary tomatoes.

After introducing me to his constant companion, Charlie (a slobberingly gregarious Labrador-Rottweiler mix), Lippman walked me through hundreds of plants, coddled by 80-degree daytime temperatures and 40 to 60 percent humidity, and goaded into 14 hours of daily photosynthetic labor by high-pressure sodium lights overhead. Some were seedlings that had barely unfurled their first embryonic leaves; others had just begun to flash their telltale yellow flowers, harbingers of the fruit to come; still others were just about ripe, beginning to sag with the weight of maturing red fruit.

What makes this greenhouse differentwhat makes it arguably an epicenter of a revolution in plant biology that may forever change not just the future of the tomato but the future of many cropsis that 90 percent of the tomato plants in the building had been genetically altered using the wizardly new gene-editing tool known as Crispr/Cas-9. Lippman and Joyce Van Eck, his longtime collaborator at the Boyce Thompson Institute in Ithaca, New York, are part of a small army of researchers using gene editing to turn the tomato into the laboratory mouse of plant science. In this greenhouse, Crispr is a verb, every plant is an experiment, and mutant isnt a dirty word.

Lippman walked to the rear of the building and pointed out a variety of tomato known as Large Fruited Fresh Marketone of the commercial varieties that turn up in supermarkets, not farmers markets. This particular plant, about two months old, bowed with big, nearly ripe fruit. It was, Lippman explained, a mutant called jointless. Most tomato varieties have a swollen knuckle of tissue (or joint) on the stem, just above where the fruit forms; when the tomato is ready, it tells itself, as Lippman put it, OK, Im ripetime to fall, and the cells in the joint receive a signal to die, letting go of the tomato. That is natures way of spreading tomato seeds, but the joint has been a thorny problem for agricultural production, because it leaves a residual stem that pokes holes in mechanically harvested fruit. Jointless tomatoes, whose stems can be plucked clean, have been bred and grown commercially, but often with unwanted side effects; these gene-edited versions avoid the unintended consequences of traditional breeding. We can now use Crispr to go in and directly target that gene for the molecular scissors to cut, which leads to a mutation, Lippman said. Voil: the jointless trait in any variety you want.

We moved on to several examples of Physalis pruinosa, a relative of the tomatillo that produces a small, succulent fruit called a ground cherry. The plant has never been domesticated, and Lippman referred to the wild version as a monstrosity: tall, unkempt, and stingy, bestowing a single measly fruit per shoot. Next to it stood a Physalis plant after scientists had induced a mutation called self-pruning. It was half as tall, much less bushy, and boasted half a dozen fruits per shoot. Lippman plucked a ground cherry off one of the mutated plants and offered it to me.

Smell it first, he entreated. Enjoy the smell. It was exotic and faintly tropical. I popped it in my mouth and bit into a complex burst of flavor. Like all its cousin tomatoes, the taste was a mystical, time-lapse blur of sugar and acidity, embellished by the whiff of volatile compounds that found my nose and rounded out the flavor.

You just ate an edited plant, Lippman said with a smile. But dont be too nervous.

Zach Lippman in a Cold Spring Harbor test field of tomato plants edited to produce more fruit.

Dolly Faibyshev

A gene-edited tomato plant.

Dolly Faibyshev

Like the majority of scientists, Lippman regards genetically modified plants as safe to eat. But his mischievous smile acknowledged that not everyone views the technology as innocuous. There is a lot of nervousness about genetic tinkering with food plants. Genetically modified (GM) transgenic crops such as corn and soybeans have infiltrated processed foods, animal feed, and biofuels for many years, and the battle over them has long divided the public in the US and overseas. The Crispr revolution is reinventing, if not reigniting, that debate. Most of the plants that have been gene-edited to date have been created by knocking out genes (that is, mutating them), not by introducing genes from unrelated species, as first-generation genetic modification generally didrousing cries of Frankenfoods and fears of environmental contamination. Precisely because its subtraction rather than addition, scientists argue that this form of gene editing mimics the process of agriculturally induced mutations that characterizes traditional plant breeding. This distinction may not assuage critics, but it has apparently persuaded federal regulators; gene-edited soybean and potato crops are already in the ground, and last March the US Department of Agriculture declared that crops developed with gene-edited mutations are indistinguishable from those produced by traditional breeding and do not require regulatory oversight.

Huge questions vex the future of foodhow to feed 9 billion mouths, how to farm in an era of unprecedented climate uncertainty, how to create more resilient and nutritious foods for a public wary of the new technology. Plant scientists are already using Crispr and related technologies to reshape food crops in dramatic waysediting wheat to reduce gluten, editing soybeans to produce a healthier oil, editing corn to produce higher yields, editing potatoes to store better (and not throw off a carcinogen when cooked). In both industrial and academic labs, new editing tools are being developed that will have a profound impact on the foods all of us eat. Yet this newfound power to transform food traits coincides with a moment when the agriculture business has consolidated into essentially three mega-conglomerates. Those companies have the money to put this new technology to use. The question is: What use will they put it toward?

Soybeans, potatoes, and corn melt invisibly into the food chain, but tomatoes add a big red exclamation point to the current debate. Perhaps no food crop is more emblematic of what is at stakeagriculturally, biologically, culturally, and perhaps even in homegrown foodie waysthan the tomato: queen of the farmers market, jewel of the backyard garden, alpha vegetable of locavores everywhere. Lippmans greenhouse reveals just some of the ways gene editing is already altering the tomatohe has plants that flower earlier, that are oblivious to daylight cues, that prune themselves into smaller footprints, that can be genetically programmed to space out the position of fruits on the stem like an accordion.

For people who love to eat or grow tomatoes (I do both), the arrival of Crispr provokes both cynicism and giddy hope about the future of our favorite vegetable. Cynicism because most of the practical scientific efforts would perpetuate the dreary taste of commercially produced tomatoes. In one sense, this is simply the latest in a century-long conquest of the produce aisles by the desires of food growers, who prize greater yield at lesser cost, over the desires of consumers, who cherish taste and nutrition. (Harry Klee, a tomato expert at the University of Florida, says that the perfect tomato for industry is one that exactly matches the size of a McDonalds hamburger.) Hope because there is something intriguing about using new technology to preserve the ravishing, sweet acidic burst of an heirloom tomato in a hardier, disease-resistant plantan heirloom-plus, if you will.

After Lippman walked me through his garden of man-made mutations, I couldnt resist asking if the heirlooms I struggle to grow every year might also benefit from Crisprs scissors.

Were not doing any editing of heirlooms, Lippman said. Not yet. But its in the works. They could benefit from a little bit of tweaking.

Tomatoes are coddled and goaded into photosynthetic labor in a greenhouse at the Boyce Thompson Institute.

Dolly Faibyshev

This is a story about tomatoes, of course. But it is also, like all agricultural stories, about mutationsnatural mutations and man-made mutations, invisibly insidious mutations and overtly grotesque mutations, mutations that were created earlier this year at Cold Spring Harbor Laboratory and mutations that may have occurred 10,000 years ago, like the ones that transformed Solanum pimpinellifolium from a scraggly perennial weed producing pea-sized fruit along the Pacific coastal margins of Peru and Ecuador to those beautiful big-lobed heirlooms in your backyard. Our cultural thesaurus has reduced the word mutant to a term of derision, but if you think mutation is a dirty word, you should probably stop readingand probably stop eating plant-based food too. The foundational principle of plant breeding is to take advantage of genetic modification, whether the mutation is caused by sunlight or x-rays or Crispr. As Klee puts it, there isnt a single crop that I know of in your produce aisle that is not drastically modified from what is out there in the wild.

Every backyard gardener is a connoisseur, witting or otherwise, of mutation. The intense, thin-skinned freshness of Brandywines, the apricot glow of Jaune Flamme, the green standoffish shoulders of Black Krims, and my personal favorite, Rose de Berne, with its blush of color and amazing tasteall those heirlooms are the product of long-ago, hand-me-down mutations.

Every spring, almost inevitably during March Madness (this year, during Villanova-Michigan), I get down on the floor with a bunch of peat pots and starter soil and clumsily press seeds of all of the above varieties into virgin dirt. My wife wonders why I cant buy seedlings at the market like everyone else, but Ive never outgrown the childlike thrill of watching an itty-bitty snippet of plant DNA, encased in the stiff callus of a seed coat, unfurl into a 5-foot-tall plant that yields its sublime bounty. Gardenersthe original DIY biologistsall know this thrill. And so does Lippman. Thats how he got into gene-editing tomatoes in the first place.

If you think mutation is a dirty word, you should probably stop reading. And probably stop eating plant-based food too.

Lippman grew up in Milford, Connecticut; his father was an English teacher and his mother worked in health care. Among his earliest memories is visiting a nearby farm with his father when he was 6 or 7 years old and picking up leftover Halloween pumpkins and gourdswith their mind-blowing shapes and colorsthat littered the field.

That pumpkin field was part of Robert Treat Farm, and when he was 13, Lippman began working summers there, cultivating his fascination with plants. By the time he graduated from high school in 1996, he had decided to pursue plant breeding and genetics, first at Cornell University and then at Cold Spring Harbor, where he got his PhD and is now a Howard Hughes Medical Institute investigator.

Lippmans office is a shrine to the tomato: On his walls are old tomato-can labels and antique postcards of implausibly gigantic tomatoes, and thousands of little brown envelopes containing seeds, each marked by year and variety, are stacked on his desk, in old seed boxes, in wooden trays and plastic cabinets against the wall. The most telling relic is just behind the door: a large framed reproduction from a 16th-century book by Pietro Andrea Mattioli, believed to be the earliest color depiction of the tomato following the Spanish conquest of the Americas. To a geneticist like Lippman, the Mattioli print is especially significant because it is early evidence that pre-Columbian cultures knew a beneficial tomato mutation when they saw onethey had already converted the nubbin of wild fruit into a large, multiple-lobed golden beefsteak.

Up until the 1930s, agricultural scientists essentially relied on the same techniques as the original tomato farmers in Central America: Be patient enough to wait for nature to produce a useful mutation, be smart enough to recognize that desirable trait (bigger fruit, for example), and be clever enough to create a new variety with that trait by selecting the mutant strains and propagating them. Put another way, agriculture has always been about unnatural selectionhuman choice privileging certain mutations while discarding others. Biologists sped up this process around the time of World War II by deliberately inducing random mutations in seeds with the use of chemicals, x-rays, and other forms of radiation. But even so, the process was slow. Selective breeding of a desirable trait could easily take a decade.

This all began to change in 2012, an annus mirabilis for the tomato. In May of that year, plant geneticists completed the Tomato Genome Projectthe entire DNA sequence of the tomato plant, all 900 million base pairs on 12 chromosomes. Then, in June, a group led by Jennifer Doudna at UC Berkeley published the first report on the new gene-editing technique known as Crispr, followed soon after by a group at the Broad Institute of MIT and Harvard. The fruit of those two converging streams of researchand, yes, botanically speaking, tomato is a fruitwas a race among scientists to see if the new technique worked in plants.

As soon as word of Crispr broke, Lippman wondered, Can we do it in tomato? And if we can, lets move. Moving fast meant doing an experiment on a tomato gene that would prove the efficacy of Crispr without too much delay. Which gene did Lippman and Van Eck target? Not one that would improve the size or shape of the fruitthat would take too long, and Van Eck was impatient. I dont want to have to put it in the greenhouse and wait for it to grow, she told Lippman. I want to be able to see something in the petri plate. So they picked a gene that was of zero economic significance and less-than-zero consumer appeal. It was a weird gene that, when mutated, produced disfigured tomato leaves that looked like needles. The mutant version was called wiry.

A research field at Cold Spring Harbor with some 8,000 gene-edited plants.

Dolly Faibyshev

Seeds are stored in boxes, and then planted.

Dolly Faibyshev

The wiry mutation was so obscure that Van Eck had to dig up a paper from 1928 that described it for the first time to know what shed be looking for. Each Crispr-directed mutation requires a customized, genetically engineered tool called a constructa so-called guide RNA to target the right tomato gene and an enzyme riding shotgun to cut the plant DNA at precisely that spot. In this case, Lippman designed the construct to target the wiry gene and cut it; the mutation is not created by Crispr per se but by the plant when it attempts to repair the wound. Van Eck used a bacterium that is very good at infecting plants to carry the Crispr mutation tool inside tomato cells. Once mutated, these cells were spread onto petri plates where they began to develop into plants. Van Eck still had to wait about two months before the tomato cells developed into seedlings and sprouted leaves, but it was worth the wait.

I still remember when I saw the first leaves coming up, she recalls. The leaves were radializedcurled up into needlelike shapes. Omigod, it worked! she cried, and raced down the hallways of the institute to tell anyone who would listen. I was thrilled because, you know, when does something work the first time?

Not only had they demonstrated that Crispr could produce a heritable trait change in a fruit crop, they also had their answer in two months rather than a year. They knew that the same basic process could theoretically be used to edit, with exquisite precision and unprecedented speed, any gene in any food crop.

As soon as they knew it worked, Lippman and Van Eck began Crispring every trait theyd wanted to study for the past 15 years. One of them was jointless. For 60 years, researchers had been trying to solve the problem of the joint on the tomato plants stem. Large-scale farming of tomatoesCalifornia alone produces more than 10 million tons each yearrequires mechanical harvesting, and those stabbing stems of jointed tomatoes make the task harder and more wasteful. Lippman, who studies plant architecture, knew that many jointless tomato plants produce excessive branching and lower yields. He discovered that this unintended consequence was the result of traditional breeding: When breeders favored the jointless mutation, they unwittingly produced unwanted branching as well because of a complex interaction between jointless and another ancient mutation. Traditional breeding produced another side effectabnormally shaped tomatoesbecause the process of selecting the jointless trait dragged along a chunk of DNA with an unwanted mutation. (This phenomenon is known as linkage drag.)

If Lippman could Crispr his way to the jointless mutation without dragging along the deleterious effects related to traditional breeding, it would offer a significant advance for growers. He and Van Eck had to wait longer than they had for the needle-nosed leaves of wiry, but by March 2016, Lippman had jointless tomatoes growing in his greenhouse. They published the work in the journal Cell in the spring of 2017, and Lippman shared the gene-editing tool with Klee at the University of Florida. Last March, Klee and his team planted a plot of gene-edited jointless mutants, in a commercial variety called Florida 8059, in a test field north of Gainesville.

Joyce Van Eck saw curled leaves on a tiny tomato plant growing on a petri plate and knew that the Crispr experiment was a success.

Dolly Faibyshev

Quick reality check: Despite the hype about the gene-editing revolution, the past couple of years have revealed limitations as well as successes. Scientists will tell you Crispr is great at knocking out a gene. But using it to insert a new gene and, as many popular accounts suggest, rewrite the germline of man, beast, or plant? Not so easy. Crispr is not the be-all and end-all, says Dan Voytas of the University of Minnesota, one of the pioneers of agricultural gene editing. Moreover, genomes are complex, even in plants. Just as a dozen knobs on a stereo console can shape the overall sound of a single song, multiple genetic elements can control the effect of a single gene.

That daunting complexity inspired Lippmans lab to pursue a clever riff on gene editing. I remember having a sticky note here, Lippman says, pointing to his keyboard. The note simply read: Promoter CRISPR.

In plants as well as animals (and humans), there is part of the DNA that lies outside the protein-encoding segment of the gene and essentially regulates its output. This upstream patch of regulatory DNA is called the promoter, and it sets different levels of outputvolume, if you willfor specific genes, from a little to a lot. What if, Lippmans group asked, you could use Crispr to, in effect, adjust the volume of a particular gene, turning it up or down like a stereo knob, by mutating the promoter in different places?

The Long Island greenhouse is now full of examples of what happens. As they reported in Cell last October, Daniel Rodrguez-Leal and colleagues in Lippmans lab showed that, by mutating the promoter of the self-pruning gene in different places, they could adjust its output like a dimmer switch, producing subtle but important changes. By using Crispr to create varying doses of a gene, Lippman says, scientists can now find better versions of plants than nature ever provided.

But better for whom? One of Lippmans pet phrases is sweet spotthat point of genetic balance where desirable traits for agriculture can be improved without sacrificing essential features like flavor or shape. Now we can start to think about taking some of our best tomato varieties, and if they can flower faster, you can start to grow them in more northern latitudes, where the summers are shorter, he says. We can begin to imagine new crops, or new versions of existing crops, for urban agriculture, like tiered cropping that they have in these abandoned warehouses ... Adapt the plant so that its more compact, flowers faster, gives you a nice-sized fruit with a decent yield, in a very compressed growth setting, with the equivalent of protective agriculturegreenhouse conditionsbut with LED lights. Because every plant gene comes with its own promoter, this genetic tuning, as Lippman puts it, could apply to virtually any vegetable crop.

The sad reality is that industry is not really committed to making a better-tasting tomato.

Tuning is just one of many ways biologists are remaking the tomato. Last year, researchers at the Sainsbury Laboratory in England gene-edited a tomato variety called Moneymaker to be resistant to powdery mildew, and a Japanese research group recently created tomatoes without seeds. On the day in May that I set my first heirloom seedlings into the ground, I happened to have a Skype conversation with two plant biologists in Brazil who have taken the gene editing of tomatoes to a whole new level. In collaboration with the Voytas lab at the University of Minnesota, Agustin Zsgn of the University of Viosa and Lzaro Peres of the University of So Paulo claim to have, in essence, reverse-engineered the weedlike wild tomato believed to be the forerunner of all cultivated varieties. (They havent published this work to date, but have discussed it at meetings.) Rather than tweak a domesticated variety of tomato, they went back to square onethe wild plantand used Crispr to knock out a handful of genes all at once. The result? Where the wild plant was sprawling and weedy, the gene-edited tomato was compact and bushy; where the ancestral plant had pea-sized fruit, the gene-edited version had reasonably plump, cherry-sized tomatoes. The edited fruit also contained more lycopene, an important antioxidant, than any other known variety of tomato. The process is called de novo domestication.

We didnt go from pea-sized to beefsteak, but we went from pea-sized to cherry-sized, said Zsgn of this first attempt. And how did the tomatoes taste? They taste great! Peres insisted. In a similar vein, Lippman and Van Eck are domesticating the wild ground cherry in the hope that it can join blueberries and strawberries as one of the basic berry crops.

What makes the de novo approach so intriguing is that it takes advantage of all the accumulated botanical wisdom of a wild plant. Over tens of thousands of years of evolution, a wild species acquires traits of hardiness and resilience, such as resistance to disease and stress. Domestication eliminated some of those traits. Since those resistance traits typically involve a suite of genes, Peres says, they would be extremely difficult to introduce into domesticated tomatoes, via Crispr or any other technology. And the approach can exploit other extreme traits. Peres wants to domesticate a wild species from the Galapagos, which can tolerate extreme environmental conditions such as high salinity and droughttraits that might enhance food security in a future with enormous climate fluctuations.

Rising temperatures. Changing growing seasons. A rising global population. The environmental toll of herbicide overuse. What if gene editing, for example, could favor disease-resistance genes that would reduce pesticide use? Lippman asks. Thats not just feeding the world, thats protecting the planet.

Lippman, outside a tomato greenhouse: Ive eaten many gene-edited tomatoes, yeah.

Dolly Faibyshev

All this new plant scienceknocking out genes, fiddling with the volume knob of promoters, de novo domesticationis wonderfully creative and happening very fast. But sooner or later, the other shoe drops in the conversation. Will consumers want to eat these tomatoes? Are Crispr vegetables and grains simply new GMOs, as a number of environmental groups maintain, or are gene-edited plants intrinsically different? This is the beginning of the new conversation, Lippman says.

The old conversation was acrimonious and emotional. The initial GM foods that Monsanto introduced in the 1990s were transgenic, meaning that biologists used genetic engineering to introduce foreign DNA, from an unrelated species, into the plant. Gene editing is much more analogous to older forms of mutagenesis such as irradiation and chemicals, though much less scattershot. Rather than creating random mutations, Crispr targets specific genes. (Editing that misses its mark is possible, though Lippman hasnt detected any in his work.) That is why plant scientists have been so eager to use it, and why the USDA regards gene-edited knockouts as similar to earlier mutagens and thus not requiring special regulation. (In the case of knocking in, or adding, a gene to crop plants, the USDA has indicated it will assess on a case-by-case basis.) Some European countries have banned GMOs, and the European Union has yet to issue a final judgment on gene-edited plants.

Although multiple studies have failed to show that GMOs pose a threat to human health, public doubts persista Pew Research Center survey in 2016 indicated that 39 percent of Americans believe that genetically modified foods are less healthy than non-GMOs, and in his household, Lippman admits, his wife initially preferred not to eat his gene-edited tomatoes.

The domesticated tomato possesses thousands, perhaps millions, of spontaneous mutations that helped turn a forlorn, ground-hugging weed into the most popular American garden plant. Now scientists are using gene editing to create these mutations and optimize the plants.

self-pruningThis mutation affects the plants size, shape, and compactness and alone can change the wild, sprawling shrub into the orderly, compact crop familiar to gardeners.

self-pruning 5gThis affects the tomatos day-length sensitivity apparatus, allowing it to be grown during shorter summers at northern latitudes.

fasciatedThis mutation affects the plants size, shape, and compactness and alone can change the wild, sprawling shrub into the orderly, compact crop familiar to gardeners.

jointless-2This mutation eliminates a break point in the middle of the stem, just above the fruit, facilitating mechanical harvesting.

lycopene beta-cyclaseThis mutation increases the fruits lycopene, the chemical that gives the tomato its red color.

There are other reasons that genetically altered foods continue to arouse suspicion. Monsantos early GMO effort used a revolutionary technology not to make healthier or more environmentally sustainable foods but to confer resistance in soybean and corn to the companys proprietary herbicide, Roundup. The companys aggressive promotion of such a self-serving first product was considered a public relations disaster.

Big agribusinesses are now positioning themselves to take advantage of gene editing. A recent rash of mergers has created three giant multinationals in global agriculture: Bayer (which completed its acquisition of Monsanto this year), DowDuPont (following Duponts earlier merger with Dow Chemical), and Syngenta (which was acquired last year by the huge Chinese gene-editing company ChemChina). The intellectual property issues are possibly more complex than plant genetics. Both the Broad Institute and DuPont Pioneer hold basic Crispr patents that apply to agriculture, and the two entities teamed up last fall to jointly negotiate licenses for farming applications (all three giant agribusinesses have licensed the technology). According to agricultural sources, the right to use Crispr for commercial agriculture requires an upfront fee, annual royalty payments on sales, and other conditions. (The Broad Institute did not discuss licensing terms, except to say that it is not involved in product development.)

This is where gene editing bumps up against the harsh economics of agriculture. Academic scientists can conduct basic research with Crispr without paying a licensing fee. But thats as far as it goes. I cant develop products and start to sell them, Lippman says. Commercial development requires payment of a licensing feea cost more easily borne by deep-pocketed agricultural companies.

There are some smaller biotechs seeking to maneuver around the giant companies and the intellectual property obstacles. Calyxt, a Minnesota-based firm cofounded by Voytas, has already received USDA approval to grow several crops using an earlier and more-difficult-to-use gene-editing technology known as TALENs. Lippman consults for a Massachusetts startup called Inari. Benson Hill Biosystems, based in St. Louis, has been working on improving plant productivity using a patented set of new gene-editing scissors the company calls Crispr 3.0. But CEO Matthew Crisp (yes, thats his name) claims innovation is being stifled by an intellectual property landscape that is very murky. Benson Hills partners and prospective licensees, he says, have complained that commercial rights to Crispr gene-editing technology are too expensive, too cumbersome, or too uncertain. The discovery of new gene-editing enzymes and other innovations may complicate the patent landscape even more. As one source put it, Its a mess. And its only going to get worse.

Thats why theres a lot of attention focused on a new startup called Pairwise Plants, in which Monsanto has teamed up with several Crispr pioneers from the Broad Institute. Recent statements to Bloomberg by company CEO Tom Adams, a former vice president of Monsanto, stressing new crops that are really beneficial to people, raised some eyebrows. You know, its not Monsanto language, Voytas noted. And the Monsanto pedigree has some plant biologists concerned. The question will be: They have enormous baggage in terms of consumer acceptance, Lippman says. And if they botch it, theyre going to ruin it for everybody else. Everyone is sort of holding their breath.

Trays for germinating tomato seeds.

Dolly Faibyshev

Physalis pruinosa plants at the Boyce Thompson Institute in Ithaca.

Dolly Faibyshev

Heres a simpler question: What about flavor? When I asked Harry Klee if he had tasted any of the jointless 8059 tomatoes hes growing, he laughed and said he hadnt bothered. We know that Florida 8059 by itself doesnt really have too much taste to begin with. A better-tasting tomato always plays second fiddle to market economics. The majority of tomatoes grown in Florida, for example, go to the food service industryto the McDonalds and Subways, Klee says. The sad reality, Klee says, is that industry is not really committed to making a better-tasting tomato. Klee loves to talk about tastehe heads a group that identified about two dozen genetic regions related to exceptional tomato flavor. We know exactly how to give you a sweeter tomato that will taste better, he says. But those tomatoes are not as economically attractive to producers. The growers wont accept it.

What about consumers? Would they accept a gene-edited tomato if it tasted better? Or, to put it in a slightly more idiosyncratic way, would it be botanical blasphemy to gene-edit an heirloom?

In his tour of the greenhouse, Lippman paused at one point to express good-natured scorn for heirlooms. They are terrific tomatoes, he admits, but pretty crappy producers. From personal experience, I can confirm that heirlooms are finicky plants and stingy producers, with lousy immune systemsmost of all, theyre heartbreakers, at least in the backyard. They start out like Usain Bolt in the 100 meters and end up looking spent, shriveled, hobbled by all manner of wilts and fungi and pests, leaves drooping like brown funereal crepe. It was tempting to think of using the new genetic tools to improve them. Klee is very anxious to introduce gene editing into the home garden. He thinks gardeners like me might be the place to make the argument that gene-edited tomatoes are not GMOs.

What if I could give you a Brandywine that had high lycopene, longer shelf life, and was a more compact plant? Klee asked me. I could do all of those today, with knocking out genes and genome editing. And I could give you something that was virtually identical to Brandywine that was half as tall and had fruit that didnt soften in less than a day, and were deep, deep red with high lycopene. I mean, would you grow that?

Absolutely! I told him.

I think most people would grow that, he replied. I think this could be a huge opportunity to educate home gardeners in what plant breeding is all about.

Not everyone would agree with Klee (or me). Voytas, a pioneer of gene editing in plants, chuckled when I asked him about a gene-edited heirloom. You know, part of it is, theyre heirloom, he said. The name inherently suggests this is something of value from the past. Not something new and techy. More to the point, he reminded me of the sort of outrageous licensing fees for the gene-editing technology. So your heirloom tomato idea would never be financially lucrative enough to pay the requisite licensing fees.

The bottom line: Gene-edited tomatoes are probably on their way to the market. But tomatoes with better flavor? Probably not going to happen anytime soon.

In early June, Zach Lippman went back to being a farmer. On what initially seemed like a sunny day, he and a dozen coworkers got their hands dirty transplanting some 8,000 gene-edited tomato plants into an outdoor field on the grounds of Cold Spring Harbor Laboratory. There were lots of the familiar mutantsjointless, self-pruning, daylight insensitivity. (The outdoor planting required prior approval from the USDA.) Plant em deep! he cried, as the crew raced to get the tomato seedlings in the ground under suddenly darkening skies.

The ultimate fate of the gene-edited tomato is as unpredictable as the weather, but the fate of these particular tomatoes is less of a mystery. Lippman often takes them home. Ive eaten many gene-edited tomatoes, yeah, he laughs. (Not surprisingly, he finds absolutely nothing different about them.) Theyre not GMO, he insists. Its just that youre left with what would be equivalent to a natural mutation. So why not eat it? Its one of just thousands or millions of mutations that may or may not affect the health of the plant and were still eating them!

Stephen S. Hall is the author of six books and teaches science writing at New York University.

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Hemophilia Gene Therapy Market 2019 to Showing Impressive …

Apr 12, 2019 (The Expresswire via COMTEX) -- Hemophilia Gene Therapy Market report is a complete study of current trends in the Market, industry growth drivers, and restraints. It provides Market forecasts for the coming years

Global Hemophilia Gene Therapy Market report observes different predilections, obstructions, and difficulties looked by the best makers/Economy by Business Leaders contenders of Complete Reports Hemophilia Gene Therapy market. Our specialists' group has considered every single angle directly from the piece of the pie, size, status, and development.

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Synopsis of the Hemophilia Gene Therapy Market: -

Hemophilia Gene Therapymarket competition by top manufacturers/ Key player/ Economy by Business Leaders: Spark Therapeutics, Ultragenyx, Shire PLC, Sangamo Therapeutics, Bioverativ, BioMarin, uniQure, Freeline Therapeutics,. And More

Hemophilia is a rare bleeding disorder in which the blood does not clot normally. Hemophilia is a monogenic disease (a disease that is caused by a genetic defect in a single gene). There are two types of hemophilia caused by mutations in genes that encode protein factors which help the blood clot and stop bleeding when blood vessels are injured. Individuals with hemophilia experience bleeding episodes after injuries and spontaneous bleeding episodes that often lead to joint disease such as arthritis. The most frequent forms of hemophilia affect males.

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The next part also sheds light on the gap between supply and consumption. Apart from the mentioned information,growth rateof Hemophilia Gene Therapy market in 2024is also explained.Additionally, type wise and application wise consumptiontables andfiguresof Hemophilia Gene Therapy marketare also given.

Table of Contents

Market Overview 1.1 Hemophilia Gene Therapy Introduction 1.2 Market Analysis by Type 1.3 Market Analysis by Applications 1.4 Market Analysis by Regions 1.4.1 North America (United States, Canada and Mexico) 1.4.1.1 United States Market States and Outlook (2013-2023) 1.4.1.2 Canada Market States and Outlook (2013-2023) 1.4.1.3 Mexico Market States and Outlook (2013-2023) 1.4.2 Europe (Germany, France, UK, Russia and Italy) 1.4.2.1 Germany Market States and Outlook (2013-2023) 1.4.2.2 France Market States and Outlook (2013-2023) 1.4.2.3 UK Market States and Outlook (2013-2023) 1.4.2.4 Russia Market States and Outlook (2013-2023) 1.4.2.5 Italy Market States and Outlook (2013-2023) 1.4.3 Asia-Pacific (China, Japan, Korea, India and Southeast Asia) 1.4.3.1 China Market States and Outlook (2013-2023) 1.4.3.2 Japan Market States and Outlook (2013-2023) 1.4.3.3 Korea Market States and Outlook (2013-2023) 1.4.3.4 India Market States and Outlook (2013-2023) 1.4.3.5 Southeast Asia Market States and Outlook (2013-2023) 1.4.4 South America, Middle East and Africa 1.4.4.1 Brazil Market States and Outlook (2013-2023) 1.4.4.2 Egypt Market States and Outlook (2013-2023) 1.4.4.3 Saudi Arabia Market States and Outlook (2013-2023) 1.4.4.4 South Africa Market States and Outlook (2013-2023) 1.4.4.5 Nigeria Market States and Outlook (2013-2023) 1.5 Market Dynamics 1.5.1 Market Opportunities 1.5.2 Market Risk 1.5.3 Market Driving Force 2 Manufacturers Profiles

3 Global Hemophilia Gene Therapy Market Analysis by Regions

4 Global Hemophilia Gene Therapy Market Competition, by Manufacturer

5 Sales Channel, Distributors, Traders and Dealers

Continued

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Stem Cell Treatment Cardiovascular Disease, Heart Disease …

Cardiovascular disease, also called heart disease, is a broad medical term used to describe a group of conditions that affect the blood vessels or the heart. It is the most common cause of death worldwide.1

Conditions of cardiovascular disease include:

The Stem Cells Transplant Institutein Costa Rica, uses adult autologous stem cells for the treatment of cardiovascular disease (heart disease). The symptoms of cardiovascular disease will depend on the specific type of heart disease.

Treatment at the Stem Cells Transplant Institute could help improve the symptoms of cardiovascular disease such as:

Heart disease and cardiovascular disease are often used interchangeably. These terms refer to a group of conditions that affect the blood vessels and heart. Valvular heart disease affects how the valves pump blood flow in and out of the heart. Cardiomyopathy affects the contractions of the heart muscle. Heart arrhythmias are disturbances in the electrical conduction making the heart beat irregular. Coronary artery disease is the most common cause of cardiovascular disease and stem cell therapy may be an effective treatment.

Coronary artery disease is caused by atherosclerosis, the buildup of plaque, causing a narrowing or blocking the blood vessels in the coronary arteries. Coronary artery disease is the leading cause of cardiovascular disease. Atherosclerosis can lead to chest pain, heart attack or stroke.

Coronary arteries carry oxygen rich blood to the heart. Plaque is caused by the presence of cholesterol, calcium, fat, and other substances in the blood. When plaque builds up in the blood vessels it narrows the arteries causing them to harden and weaken, reducing the amount of oxygen rich blood to the heart. As a result, the heart cannot pump blood effectively to the rest of the body potentially leading to heart failure and ultimately death.

If the plaque building up in the coronary arteries breaks, a blood clot forms around the plaque. If the clot cuts off the blood flow to the heart muscle completely, the heart muscle is unable to get the necessary oxygen and nutrients causing a part of the heart muscle to die. The result is a heart attack or myocardial infarction,

Coronary artery disease, high blood pressure or a previous heart attack can lead to the onset of heart failure. Heart failure is a chronic, progressive disease typically caused by another heart condition resulting in the heart muscle losing its ability to supply the rest of body with enough blood and oxygen.

Atherosclerosis can also cause peripheral artery disease. Peripheral arterial disease occurs when the narrowed peripheral arteries cannot send enough blood flow to the extremities, usually the legs. The most common symptoms of peripheral artery disease are; cramping, pain, and/or tiredness in the leg or hip muscles during exertion. The most severe symptom of peripheral artery disease is critical limb ischemia, pain at rest due to reduced blood flow to the limb.

Approximately 85% of strokes are ischemic strokes. Atherosclerosis is the most common cause of ischemic stroke. If the arteries become too narrow due to plaque buildup, the blood cells may collect and form a clot. A larger clot can block the artery where it is formed (thrombotic stroke) while a smaller clot may travel until it reaches an artery closer to the brain (embolic stroke). When the arteries to your brain become narrow or blocked, the required blood flow is reduced resulting in stroke. Other causes of ischemic stroke are clots due to an irregular heartbeat or heart attack.

Stem cell therapy at the Stem Cells Transplant Institute may be a good alternative for patients seeking a safe, non-surgical treatment for cardiovascular disease.

Notably, adult stem and progenitor cells including.mesenchymal stem cells have progressed into clinical trials and have shown positive benefits.5

Stem cell transplantation uses healthy cells to promote the repair of damaged cells and regeneration of healthy and functional cells to repair injured tissue.1 The therapeutic effect of stem cell transplantation in patients with cardiovascular disease may be due to the paracrine effect. The theory is transplanted stem cells repair damaged tissue by releasing factors that promote regeneration of healthy stem cells, reduce inflammation, promote the growth of new blood vessels, inhibit cell death, and reduce hypertrophy.1

The results of initial research using mesenchymal stem cell transplantation:

Heart Failure

Adipose derived stem cells improve left ventricular function, promote angiogenesis, lower fibrosis, and decrease inflammation. Several months following treatment, stem cells continue to migrate to the heart muscle regenerating and renewing healthy heart function. Stem cell therapy cannot help all patients with cardiovascular disease but for many patients stem cell therapy combined with lifestyle modification may be a safe, effective, non-surgical alternative treatment.

Lifestyle changes that can help improve cardiovascular disease include:

The Stem Cells Transplant Institute uses autologous mesenchymal stem cells for the treatment of cardiovascular disease. Autologous means the stem cells are collected from the recipient so the risk of rejection is virtually eliminated. Mesenchymal stem cells are one type of adult stem cells that are found in a variety of tissues including; adipose tissue, lung, bone marrow, and blood. Mesenchymal stem cells have several advantages over other types of stem cells; ability to migrate to sites of tissue injury, strong immunosuppressive effect, and better safety after infusion.2,3 Mesenchymal stem cells are a promising treatment for cardiovascular disease. Treatment at the Stem Cells Transplant Institute may improve the symptoms and long-term complications of cardiovascular disease.

A team of stem cell experts developed an FDA approved method and protocol for harvesting and isolating adipose derived stem cells for autologous reimplantation. The collection and use of adult stem cells does not require the destruction of embryos and for this reason, more U.S. federal funding is being spent on stem cell research.

The stem cells are administered intravenously.

Costa Rica has one of the best healthcare systems in world and is ranked among the highest for medical tourism. Using the most advanced technologies, the team of experts at The Stem Cells Transplant Institute believes in the potential of stem cell therapy for the treatment of cardiovascular disease. We are committed to providing personalized service and the highest quality of care to every patient. Contact us to see if stem cell therapy may be a treatment option for you.

1.Sun R.Advances in stem cell therapy for cardiovascular disease (Review). National Journal of Mol. Med. 38: 23-29, 2016. 2 Hare JM, Fishman JE, Gerstenblith G, DiFede Velazquez DL, Zambrano JP, Suncion VY, Tracy M, Ghersin E, Johnston PV, Brinker JA, et al: Comparison of allogeneic vs autologous bone marrow-derived mesenchymal stem cells delivered by transendocardial injection in patients with ischemic cardiomyopathy: the POSEIDON randomized trial. JAMA 308: 2369-2379, 2012.3 Miyahara Y, Nagaya N, Kataoka M, Yanagawa B, Tanaka K, Hao H, Ishino K, Ishida H, Shimizu T, Kangawa K, et al: Monolayered mesenchymal stem cells repair scarred myocardium after myocardial infarction. Nat Med 12: 459-465, 2006. 4 Mazo M, Planat-Bnard V, Abizanda G, Pelacho B, Lobon B, Gavira JJ, Peuelas I, Cemborain A, Pnicaud L, Laharrague P, et al: Transplantation of adipose derived stromal cells is associated with functional improvement in a rat model of chronic myocardial infarction. Eur J Heart Fail 10: 454-462, 2008. 5 Stem cell-based therapies to promote angiogenesis in ischemic cardiovascular disease Luqia Hou,1,2 Am J Physiol Heart Circ Physiol 310: H455H465, 2016. 6 Kinnaird T, Stabile E, Burnett MS, Lee CW, Barr S, Fuchs S, Epstein SE. Marrow-derived stromal cells express genes encoding a broad spectrum of arteriogenic cytokines and promote in vitro and in vivo arteriogenesis through paracrine mechanisms. Circ Res 94: 678685, 2004. 7 Kinnaird T, Stabile E, Burnett MS, Shou M, Lee CW, Barr S, Fuchs S, Epstein SE. Local delivery of marrow-derived stromal cells augments collateral perfusion through paracrine mechanisms. Circulation 109: 15431549, 2004.

8 Hare JM, Fishman JE, Gerstenblith G, DiFede Velazquez DL, Zambrano JP, Suncion VY, Tracy M, Ghersin E, Johnston PV, Brinker JA, Breton E, Davis-Sproul J, Schulman IH, Byrnes J, Mendizabal AM, Lowery MH, Rouy D, Altman P, Wong Po Foo C, Ruiz P, Amador A, Da Silva J, McNiece IK, Heldman AW, George R, Lardo A. Comparison of allogeneic vs autologous bone marrowderived mesenchymal stem cells delivered by transendocardial injection in patients with ischemic cardiomyopathy: the POSEIDON randomized trial. JAMA 308: 23692379, 2012.

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Autologous iPS cell therapy for Macular Degeneration: From bench-to-bedside

Presented At:Gibco - 24 Hours of Stem Cells Virtual Event

Presented By:Kapil Bharti - Stadtman Investigator, NIH, Unit on Ocular Stem Cell & Translational Research

Speaker Biography:Dr. Kapil Bharti holds a bachelor's degree in Biophysics from the Panjab University, Chandigarh, India, a master's degree in biotechnology from the M.S. Rao University, Baroda, India, and a diploma in molecular cell biology from Johann Wolfgang Goethe University, Frankfurt, Germany. He obtained his Ph.D. from the same institution, graduating summa cum laude. His Ph.D. work involved research in the areas of heat stress, chaperones, and epigenetics.

Webinar:Autologous iPS cell therapy for Macular Degeneration: From bench-to-bedside

Webinar Abstract:Induced pluripotent stem (iPS) cells are a promising source of personalized therapy. These cells can provide immune-compatible autologous replacement tissue for the treatment of potentially all degenerative diseases. We are preparing a phase I clinical trial using iPS cell derived ocular tissue to treat age-related macular degeneration (AMD), one of the leading blinding diseases in the US. AMD is caused by the progressive degeneration of retinal pigment epithelium (RPE), a monolayer tissue that maintains vision by maintaining photoreceptor function and survival. Combining developmental biology with tissue engineering we have developed clinical-grade iPS cell derived RPE-patch on a biodegradable scaffold. This patch performs key RPE functions like phagocytosis of photoreceptor outer segments, ability to transport water from apical to basal side, and the ability to secrete cytokines in a polarized fashion. We confirmed the safety and efficacy of this replacement patch in animal models as part of a Phase I Investigational New Drug (IND)-application. Approval of this IND application will lead to transplantation of autologous iPS cell derived RPE-patch in patients with the advanced stage of AMD. Success of NEI autologous cell therapy project will help leverage other iPS cell-based trials making personalized cell therapy a common medical practice.

LabRoots on Social:Facebook: https://www.facebook.com/LabRootsIncTwitter: https://twitter.com/LabRoots LinkedIn: https://www.linkedin.com/company/labr... Instagram: https://www.instagram.com/labrootsinc Pinterest: https://www.pinterest.com/labroots/ SnapChat: labroots_inc

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Autologous iPS cell therapy for Macular Degeneration: From bench-to-bedside

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Is Male Infertility Genetic? | Hereditary Fertility Issues …

Why does it matter if infertility has a genetic cause?

Developed in the early 1990s, assisted reproduction in the form of IVF and ICSI (intracytoplasmic sperm injection) is a revolutionary laboratory technique in which a single sperm is placed directly inside an egg for fertilization. This technique has opened the door to fertility for men who formerly had few available treatment options, as it allows men who were previously considered severely infertile or sterile the possibility of fatherhood. However, with ICSI sperm are chosen by laboratory technicians and not by nature and because of this, it is not clear what barriers to natural selection are altered. Thus, along with this technology comes the possibility of passing on to a child certain genetic issues that may have caused the fathers infertility, or even more severe conditions. Another reason to know whether male infertility is genetic or not is because classic treatments such asvaricocelerepair or medications given to improve male infertility. In fact, Dr Turek was one of the first to publishonthis issue, showing thatvaricocelerepair was not effective in improving fertility in men with genetic infertility. Because he recognized these issues early on, Dr. Turek, while at UCSF in 1997, founded the first formal genetic counseling and testing program for infertility in the U.S. Called the Program in the Genetics of Infertility (PROGENI), Dr.Tureksprogram has helped over 2000 patients at risk for genetic infertility to navigate the decision-making waters that surround this condition.

Men with infertility should be seen by a urologist for a thorough medical history, physical examination, and appropriate medical testing. If genetic infertility is a possibility, then a genetic counselor can help couples understand the possible reasons, offer appropriate genetic testing, and discuss the complex emotional and medical implications of the test results. The approach taken early on by Dr. Turek is outlined in Figure 1. Just like the medical diagnosis from a urologist or fertility specialist, information about family history plays a critical role in genetic risk assessment. This approach to genetic evaluation, termed non-prescriptive, has been the cornerstone of Dr. Tureks critically acclaimed clinical program that now has over a dozen publications contributing to our current knowledge in the field. It is important to note that a lack of family history of infertility or other medical problems does not eliminate or reduce the risk of genetic infertility. In fact, a family history review will often be unremarkable. However, family history can provide crucial supporting information toward making a genetic diagnosis (such as a family history of recurrent miscarriages or babies born with problems). Dr. Turek has published thathaving a genetic counselor obtainfamily history information is much more accurate than simply giving patients a written questionnaire to fill out and bring to their visit. A genetic counselor can also discuss appropriate genetic testing options and review the test results in patients in a meaningful way.

When speaking to Dr.Tureksgenetic counselor about genetic testing, keep in mind that he or she will not tell you what to do. Genetic counselors are trained to provide information, address questions and concerns, and support you in the decision making process. A genetic counselor does not assume which decisions are most appropriate for you.

Among the various infertility diagnoses that men have, some are more commonly associated with genetic causes. Diagnoses that can have genetic causes include men nonobstructive azoospermia (no sperm count), oligospermia (low sperm count), and congenital absence of the vas deferens. A list of some of the best- described causes of genetic male infertility and their frequencies and associated conditions are listed in Table 1.

Nonobstructiveazoospermiais defined aszero sperm countin the ejaculate due to an underlying sperm production problem within the testicles. This is quite different from obstructive azoospermia in which sperm production within the testes is normal, but there is a blockage in the reproductive tract ducts that prevents the sperm from leaving the body. There can be changes in the levels of reproductive hormones, such as follicle stimulating hormone (FSH), observed withnonobstructiveazoospermia. Most commonly, the FSH is elevated in this condition, which is an appropriate and safe hormone responseofthe pituitary gland to states of low or no sperm production. This diagnosis is associated with a 15%chance forhaving chromosome abnormalities(Figure 2) and a 13% chance for having gene regions missing on the Y chromosome (termed Y chromosome microdeletions, Figure3). To detect these changes, blood tests are typically offered to men with nonobstructive azoospermia.

Oligospermiathat places men at risk for genetic infertility occurs when the ejaculate contains a sperm concentration of

Congenital absence of the vas deferens is characterized by the malformation or absence of the ducts that allow sperm to pass from the testicles into the ejaculate and out of the body during ejaculation. The duct that is affectedinthis condition is thevasdeferens. This is the sameductthat is treated during a vasectomy, a procedure for men who want birth control. Men with this condition are essentially born with a natural vasectomy. This congenital condition is associated with mutations and/or variations in the genes for cystic fibrosis (the CFTR gene) in 70-80% men if thevasdeferensis absent on both sides, but less than this if the duct is missing on only one side. For most men with this condition with a mutation in the cystic fibrosis gene, the missingvasdeferensis the only problem that results from this genetic change and they do not have the full spectrum of symptoms associated with cystic fibrosis, the most common genetic disease in the U.S.andgenerally lethal in early adulthood.

A less common reason for mento havea zero sperm count (azoospermia) than nonobstructive azoospermia is obstructive azoospermia. In essence, this is an unexplained zero sperm count due to a blockage of the reproductive tract ducts leading from the testicle to the ejaculate. Blockages are most commonly found in theepididymisbut can also be located in thevasdeferensor ejaculatory ducts. Most cases of obstructive azoospermia are amendable to surgical repair and naturally fertility is common. However, a high proportion of these men (47%) have mutations in the cystic fibrosis gene (CFTR) or harbor variations in the CFTR gene, termed 5T alleles. As such, genetic counseling and testing is also important in these patients.

These conditions represent only the most common genetic conditions encountered when evaluating men for genetic infertility. For this reason, consider readingDr.Turekspublished paperthat discusses most of the currently understood syndromes and conditions that are associated with infertility. It is also important to remember that if all genetic test results are normal, there is still a possibility that the infertility has a genetic cause. However, in many cases, medical science is currently unable to offer testing to detect it.

If a man has a chromosome abnormality identified as the cause of infertility, then depending on the chromosome abnormality detected, there may be a higher risk for children to be born with birth defects or mental impairment. This occurs as a result of a child inheriting from the father an imbalance in chromosome material. A genetic counselor can provide more detailed information about such potential risks, and offer other resources for individuals who have been diagnosed with a chromosome abnormality. There may be support organizations available to help men with genetic diagnoses and their partners cope with the impact of this information. Some couples find it helpful to talk to others in similar circumstances.

If a man is diagnosed with a Y chromosome deletion, then he will pass on that Y chromosome deletion to anysonhe conceives. To his daughters, he will pass on his X chromosome, instead of the Y chromosome. It is assumed that any son inheriting a Y chromosome deletion from his father will also have infertility. It is unclear whether the type and severity of the infertility will be different from the fathers. So far, there have only been a few reports of sons born to fathers with Y chromosome deletions after conception by assisted reproduction. As expected, there has not been an increase in the rate of birth defects or other problems for these boys, although this group is still small in number, and too young to have fertility evaluations.

Transmission of CFTR mutations in cases of infertility due to congenital absence of the vasdeferensis somewhat more complex than either Ymicrodeletionsor a chromosome abnormality. This is because there are over 1400 described mutations in the CFTR gene and the impact of mutations differs depending on which one is present. In general, the partner of an affected man should be tested as well, so that the residual risk of a child having either congenital absence of the vas deferens or full-blown cystic fibrosis can be estimated.

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About Hormone Clinics – Hormone Clinics

Welcome to the Hormone Clinic !

At the hormone clinics we have been helping men and women to live well and achieve peak performance at any age through hormone therapy.

Our medical director, Dr. Richard Gaines was one of the pioneers in hormone therapy for men and women. He, and all of the staff with the hormone clinic possess a unique insight and decades of experience in the safe and effective use of hormone replacement therapies such as HGH Therapy,Testosterone Therapy and Bio-Identical Hormone Therapy.

We use hormone therapy to give you back what time and nature can take away.

The hormone clinic takes a very different approach to hormone therapy than you will find at Cenegenics, or any other provider of hormone therapy. At the hormone clinic you will always be treated as an individual.

We tailor your hormone therapy to your unique needs and lifestyle. Beyond that, we incorporate your hormone therapy into a program of Holistic Health and Wellness.

It is an approach to hormone therapy that is designed to help you get the most out of your treatments, in mind, body and spirit.

During your hormone therapy, you will be assigned one of our Holistic Wellness coaches. He or she will work with you to design a program of fitness, diet, stress reduction and exercise that will help you to maximize, and maintain the benefits of your hormone therapy.

You will also find the cost of hormone therapy more reasonable at the hormone clinic than you would at most other providers of hormone therapy. This is not only because of our precise and individualized dosing. We have developed long-standing relationships with certified local compounding pharmacies, which helps us to keep the costs of our bioidentical hormones low.

Also, unlike some other hormone centers, The Hormone Clinic will never lock you into a long term hormone therapy program. In addition, The Hormone Clinic will never try to sell you products or supplements along with your hormone therapy that you do not need.

All of the doctors, physicians assistants, and nurse practitioners at the Hormone Clinic are highly trained and experienced in hormone therapy. Many of them are over 45 and on the program themselves, and are running marathons, racing motorcycles, climbing mountains, and doing other great things!

The Hormone Clinic is led by well-known expert in hormone therapy Dr. Richard Gaines. For decades, Dr. Gaines has been helping men and women of any age stay young, healthy, and accomplish great things in life, by offering customized hormone therapy.

In our Miami Beach location, our hormone clinic can provide you with not only the very best in Miami hormone therapy, but is also within the building of South Floridas first integrated wellness center.

As soon as you step into any hormone clinic location, you will know immediately that you are in a unique ultra-modern facility.

At the Hormone Clinic you will be treated with the ultimate in individualized medicine. At every point of contact with our hormone therapy staff you will receive executive treatment, all delivered in a setting that is as unique as you are.

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Low Rates of Genetic Testing in Ovarian, Breast Cancer …

April 9, 2019, by NCI Staff

Many women diagnosed with ovarian and breast cancers are not receiving tests for inherited genetic mutations, according to a new study.

Credit: iStock

Tests for inherited genetic mutations can provide women diagnosed with ovarian or breast cancer with important information that can have implications for family members and potentially guide treatment decisions and longer-term screening for second cancers. However, many women with ovarian and breast cancers are not receiving these genetic tests, a new study suggests.

An NCI-funded analysis of data on more than 83,000 women from large cancer registries in California and Georgia found that, in 2013 and 2014, only about one-quarter of women with breast cancer and one-third of women with ovarian cancer underwent testing for known harmful variants in breast and ovarian cancer susceptibility genes.

The study also found that among patients who did receive genetic testing, 8% of breast cancer patients and 15% of ovarian cancer patients had actionable gene variants, meaning variants that might warrant changes in treatment, screening, and risk-reduction strategies.

The findings, published April 9, 2019, in the Journal of Clinical Oncology, were surprising, especially the low rate of testing among women with ovarian cancer, said lead author Allison Kurian, M.D., M.Sc., of Stanford University School of Medicine.

Genetic testing has become quite cheap and accessible, and this study includes a time period when it was becoming much cheaper, so its striking that we still see low rates of testing, Dr. Kurian said. I think that suggests that there are most likely other barriers outside of cost.

The study also revealed racial and socioeconomic disparities in testing rates among women diagnosed with ovarian cancer. Genetic testing rates were far lower for black women than for white women, and they were also lower for uninsured patients than for insured patients.

These findings have uncovered a [disparities] gap that is much more substantial than I would have thought, Dr. Kurian said.

About 15% of ovarian cancers are caused by inherited mutations, and several medical organizations recommend that all women diagnosed with ovarian cancer receive genetic testing.

For women with breast cancer, the recommendations for genetic counseling and testing are generally more limited, typically relying on factors such as age at cancer diagnosis and family history. However, some organizations, including the American Society of Breast Surgeons, recommend that genetic testing be made available to all women diagnosed with breast cancer.

There are many reasons why women with ovarian and breast cancer would get tested, Dr. Kurian explained.

We know that if patients have a specific inherited gene mutation, they will likely have more benefit from a new class of drugs called PARP inhibitors, she said.

The Food and Drug Administration has approved three PARP inhibitors for BRCA1-and BRCA2-associated ovarian cancer and two for BRCA1/2-associated metastatic breast cancer. Harmful variants of both BRCA1 and BRCA2 are known to increase the risk of breast and ovarian cancer, as well as of several other types of cancer.

Another reason to get tested is that patients with a genetic mutation that is associated with breast or ovarian cancer may be at higher risk of a second cancer, so you dont want to miss a second cancer that could be a problem, Dr. Kurian said.

The findings could also be life-saving information for a patients relatives. If you find that she carries a mutation, every first-degree relative, male or female, has a 50% chance of having the same mutation, she said.

Testing, then, could allow for enhanced screening and prevention for family members who are carriers, she explained.

The study included all women older than age 20 who were diagnosed with breast or ovarian cancer in California and Georgia from 20132014 and whose data were reported to NCIs Surveillance, Epidemiology and End Results (SEER) registries. There were 77,085 patients with breast cancer and 6,001 with ovarian cancer. The registry data were linked to results from four laboratories that performed nearly all the genetic testing for inherited, or germline, mutations in these states during the study period.

According to the authors, this is the first population study of hereditary cancer genetic testing in the United States with laboratory-confirmed testing results.

Weve never had this kind of linkage available before, giving us a baseline to let us know if the standard of care [for testing] was being followed, said study coauthor Lynne Penberthy, M.D., M.P.H., associate director for NCIs Surveillance Research Program. Thats why this is really important. These data can be used to see where we are and where were going. We can continue to provide this information, so people can see, hopefully, an increase in the appropriate use of genetic testing over time.

Linking the SEER registry data to the testing data in this study provides really objective data about the massive undertesting of ovarian cancer patients, said Susan Domchek, M.D., executive director of the Basser Center for BRCA at the University of Pennsylvania Abramson Cancer Center, who was not involved in the study.

Testing is recommended for all patients with ovarian cancer, she added, so the fact that only one-third of these patients had it done in this time period is a clear-cut example that were not testing ovarian cancer patients the way that we should be.

While large racial and socioeconomic disparities in testing rates were not observed among women with breast cancer, among women with ovarian cancer, testing rates were far lower in black women than white women (21.6% versus 33.8%) and in uninsured women than insured women (20.8% versus 35.3%).

Understanding why genetic testing rates are so low in women with ovarian cancer and why racial and socioeconomic disparities in testing exist among women with the disease is tricky, Dr. Kurian said.

Testing in ovarian cancer has not been widely studied beforedefinitely not at the population leveland not in such a diverse population, she added, so theres a lot we dont know about barriers.

For example, she said, its unclear whether genetic testing is on the radar screen of doctors treating patients with ovarian cancer as much as it is for patients with breast cancer. Dr. Domchek said there could also be misconceptions among patients about the costs of genetic testing.

But if access to genetic counseling or information on testing is difficult, clearing up these misconceptions can be a challenge, she said. So, trying to figure out how to better streamline [counseling and education] into practice to make sure all of these individuals with ovarian cancer get tested is a subject of ongoing research.

Dr. Domchek noted that NCI is looking to fund studies that offer genetic testing to women with a personal or family history of ovarian cancer to see if it can help to identify members of their families who may be at increased cancer risk.

Although variants in the BRCA1 and BRCA2 genes were the most frequently found in the study, the laboratories also looked for other inherited cancer-related genetic mutations using tests known as multigene panels.

The results provide an understanding, on a broader scale, of how common these mutations are, Dr. Kurian said.

The multigene panel testing led to other noteworthy findings, Dr. Penberthy said.

What was really interesting was that while BRCA1 and BRCA2 were the most common germline mutations that we found in the study, there were other mutations that were not uncommon and that were actionable in terms of treatment as well, she explained.

For example, 60 women with breast cancer in the study had a mutation in the CDH1, PALB2, or PTEN genes. These mutations are associated with a substantially increased breast cancer risk, Dr. Kurian said, so women who have these mutations may consider having both breasts removed (a risk-reducing bilateral mastectomy), rather than just the breast in which the tumor was found.

And widely used clinical guidelines recommend that women with breast cancer who have certain inherited genetic mutations,including in genes such as ATM and CHEK2,undergo more intensive screening for second cancers. In the study, mutations in ATM and CHEK2 were found in 0.7% and 1.6% of women with breast cancer, respectively.

Mutations in CHEK2 and PALB2and several other genes were found both in women with breast cancer and women with ovarian cancer. Studies havent yet linked these genes with increased ovarian cancer risk, so further study is warranted, the authors wrote.

However, the key message from this study is the undertesting of ovarian cancer patients, who clearly need it, Dr. Domchek said.

Its not to say we shouldnt debate population screening [for inherited mutations], or which genes to test for, and how were going to do it, she said. But first, for heavens sake, lets test the people who absolutely need testing, not only because it impacts family members, but also because now we have first-line therapy with PARP inhibitors. Every woman with ovarian cancer should know her BRCA1 or BRCA2 status.

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Genetic testing – Drugs.com

The originating document has been archived. We cannot confirm the completeness, accuracy and currency of the content.

Medically reviewed on Jul 19, 2018

Genetic testing involves examining your DNA, the chemical database that carries instructions for your body's functions. Genetic testing can reveal changes or alterations in your genes that may cause illness or disease.

Although genetic testing can provide important information for diagnosing, treating and preventing illness, there are limitations. For example, if you're a healthy person, a positive result from genetic testing doesn't always mean you will develop a disease. On the other hand, in some situations, a negative result doesn't guarantee that you won't have a certain disorder.

Talking to your doctor or a genetic counselor about what you will do with the results is an important step in the process of genetic testing.

Several types of genetic testing are done for different reasons:

Before you undergo genetic testing, gather as much information as you can about your family's medical history. Then, talk with your doctor or a genetic counselor about your personal and family medical history. This can help you better understand your risk. Discuss questions or concerns you have about genetic testing at that meeting. Also, talk about your options, depending on the results of the test.

If you are being tested for a genetic disorder that runs in families, you may want to consider discussing your decision to undergo genetic testing with your family. Having these conversations before testing can give you a sense of how your family might respond to your test results and how it will affect them.

Not all health insurance pays for genetic testing. So, before you have a genetic test, check with your insurance provider to see what will be covered. In the United States, the federal Genetic Information Nondiscrimination Act (GINA) helps prevent health insurers or employers from discriminating against you based on test results. Most states offer additional protection.

Your doctor, medical geneticist or nurse practitioner may administer a genetic test. Depending on the type of test, a sample of your blood, skin, amniotic fluid or other tissue will be collected and sent to a lab for analysis.

The amount of time it takes for you to receive your genetic testing results will depend on the type of test and your health care facility. Talk to your doctor before the test about when you can expect the results. The lab will likely provide the test results to your doctor in writing. Your doctor can then discuss them with you.

If the genetic test result is positive, that means the genetic alteration that was being tested for was detected. The steps you take after you receive a positive result will depend on the reason you underwent genetic testing. If the purpose was to diagnose a specific disease or condition, a positive result will help you and your doctor determine the right treatment and management plan.

If you were tested to find out if you are carrying an altered gene that could cause disease in your child, and the test is positive, your doctor or a genetic counselor can help you determine your child's risk of actually developing the disease. The test results can also provide information to consider as you and your partner make family planning decisions.

If you were having gene testing to determine if you might develop a certain disease, a positive test doesn't necessarily mean you will get that disorder. For example, having a breast cancer gene (BRCA1 or BRCA2) means you are at high risk of developing breast cancer at some point in your life, but it doesn't indicate with certainty that you will get breast cancer. However, there are some conditions, such as Huntington's disease, for which having the altered gene does indicate that the disease will eventually develop.

Talk to your doctor about what a positive result means for you. In some cases, you can make lifestyle changes that may decrease your risk of developing a disease, even if you have an altered gene that makes you more susceptible to a disorder. Results may also help you make choices related to family planning, careers and insurance coverage.

In addition, you may choose to participate in research or registries related to your genetic disorder or condition. These options may help you stay updated with new developments in prevention or treatment.

A negative result means a genetic alteration was not detected by the test. But a negative result doesn't guarantee that you don't have an alteration. The accuracy of genetic tests to detect alterations varies, depending on the condition being tested for and whether or not an alteration has been previously identified in a family member.

Even if you don't have the genetic alteration, that doesn't necessarily mean you will never get the disease. For example, people who don't have a breast cancer gene (BRCA1 or BRCA2) can still develop breast cancer. Also, genetic testing may not be able to detect all genetic defects.

In some cases, a genetic test may not be able to provide helpful information about the gene in question. Everyone has variations in the way genes appear (polymorphisms), and often, these variations don't affect your health. But sometimes it can be difficult to distinguish between a disease-causing gene alteration and a harmless gene variation. In these situations, follow-up testing may be necessary.

No matter what the results of your genetic testing, talk with your doctor or genetic counselor about questions or concerns you may have. This will help you understand what the results mean for you and your family.

Last updated: July 19th, 2013

1998-2017 Mayo Foundation for Medical Education and Research (MFMER). All rights reserved. Terms of use

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What are the types of genetic tests? – Genetics Home …

Genetic testing can provide information about a person's genes and chromosomes. Available types of testing include:

Newborn screening is used just after birth to identify genetic disorders that can be treated early in life. Millions of babies are tested each year in the United States. All states currently test infants for phenylketonuria (a genetic disorder that causes intellectual disability if left untreated) and congenital hypothyroidism (a disorder of the thyroid gland). Most states also test for other genetic disorders.

Diagnostic testing is used to identify or rule out a specific genetic or chromosomal condition. In many cases, genetic testing is used to confirm a diagnosis when a particular condition is suspected based on physical signs and symptoms. Diagnostic testing can be performed before birth or at any time during a person's life, but is not available for all genes or all genetic conditions. The results of a diagnostic test can influence a person's choices about health care and the management of the disorder.

Carrier testing is used to identify people who carry one copy of a gene mutation that, when present in two copies, causes a genetic disorder. This type of testing is offered to individuals who have a family history of a genetic disorder and to people in certain ethnic groups with an increased risk of specific genetic conditions. If both parents are tested, the test can provide information about a couple's risk of having a child with a genetic condition.

Prenatal testing is used to detect changes in a fetus's genes or chromosomes before birth. This type of testing is offered during pregnancy if there is an increased risk that the baby will have a genetic or chromosomal disorder. In some cases, prenatal testing can lessen a couple's uncertainty or help them make decisions about a pregnancy. It cannot identify all possible inherited disorders and birth defects, however.

Preimplantation testing, also called preimplantation genetic diagnosis (PGD), is a specialized technique that can reduce the risk of having a child with a particular genetic or chromosomal disorder. It is used to detect genetic changes in embryos that were created using assisted reproductive techniques such as in-vitro fertilization. In-vitro fertilization involves removing egg cells from a womans ovaries and fertilizing them with sperm cells outside the body. To perform preimplantation testing, a small number of cells are taken from these embryos and tested for certain genetic changes. Only embryos without these changes are implanted in the uterus to initiate a pregnancy.

Predictive and presymptomatic types of testing are used to detect gene mutations associated with disorders that appear after birth, often later in life. These tests can be helpful to people who have a family member with a genetic disorder, but who have no features of the disorder themselves at the time of testing. Predictive testing can identify mutations that increase a person's risk of developing disorders with a genetic basis, such as certain types of cancer. Presymptomatic testing can determine whether a person will develop a genetic disorder, such as hereditary hemochromatosis (an iron overload disorder), before any signs or symptoms appear. The results of predictive and presymptomatic testing can provide information about a persons risk of developing a specific disorder and help with making decisions about medical care.

Forensic testing uses DNA sequences to identify an individual for legal purposes. Unlike the tests described above, forensic testing is not used to detect gene mutations associated with disease. This type of testing can identify crime or catastrophe victims, rule out or implicate a crime suspect, or establish biological relationships between people (for example, paternity).

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Amazon.com: AncestryDNA: Genetic Testing Ethnicity: Health …

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Hypogonadism | California Center for Pituitary Disorders

Hypogonadism is separated into two types: primary hypogonadism (resulting from dysfunction of the testis or ovary) or central hypogonadism (resulting from pituitary or hypothalamic dysfunction that leads to loss of lutenizing horomne [LH] and follicle-stimulating hormone [FSH]).

Causes of hypogondaism include genetic, menopausual, autoimmune, viral, radiation, and chemotherapeutic agents. Central hypogonadism is often due to pituitary adenomas. Through compression of the gland, these tumors can cause destruction of pituitary tissue or interference with gonadotropin-releasing hormone (GnRH) input from the hypothalamus. Gonadotropin dysfunction is the second most common hormonal disorder from compression of the pituitary gland from a pituitary adenoma after GH suppression. Hypothalamic disorders such as tumors and hypothalamic amenorrhea, as well as exposure to radiation, can lead to hypogonadism. Fasting, weight loss, anorexia nervosa, bulimia, exercise, or stressful conditions result in defects in pulsatile GnRH secretion ("hypothalamic amenorrhea"). Elevated prolactin levels can also suppress GnRH pulses and lead to hypothalamic hypogonadism. Diagonisis requires measurement of LH, FSH, and testosterone or estrogen, with reference to age-adjusted normal values.

Hypogonadism in prepubertal children causes no symptoms, whereas in adolescents, it leads to delayed or absent sexual development.

In adult women, hypogonadism causes:

Prolonged periods of hypogonadism can cause osteoporosis.

In men, hypogonadism leads to:

Most cases of hypogonadism can be successfully treated. Treatment of hypogonadism in men and premenopausal women is effectively accomplished by replacement hormonal therapy. Fertility can be restored by administration of human chorionic gonadotropin, which acts like LH, often in combination with FSH, or by the pulsatile administration of GnRH. Treatment for hypogonadism resulting from a pituitary tumor includes surgery to remove the tumor.

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Banking Menstrual Stem Cells | What are Menstrual Stem …

Stem cells in menstrual blood have similar regenerative capabilities as thestem cells in umbilical cord blood and bone marrow. Cryo-Cell's patent-pendingmenstrual stem cell service offers women in their reproductive years the ability to store and preserve these cells for potential use by herself or a family memberfree from ethical or political controversy.

Cryo-Cell is the only stem cell bank in the world that can offer womenthe reassurance and peace of mind that comes with this opportunity.

What are menstrual stem cells?Stem cells in menstrual blood are highly proliferativeandpossess the unique ability to develop into various other types of healthy cells. During a womans menstrual cycle, these valuable stem cells are discarded.

Cryo-Cell'smenstrual stem cell bankingservice captures those self-renewing stem cells, processes and cryopreserves them for emerging cellular therapies that hold the promise of potentially treatinglife-threatening diseases.

How are menstrual stem cells collected, processed and stored?The menstrual blood is collected in a physicians officeusing a medical-grade silicone cup in place of a tampon orsanitary napkin. The sample is shipped to Cryo-Cell via a medical courier and processed in our state-of-the-art ISO Class 7 clean room.

The menstrual stem cells are stored in two cryovials that are overwrapped to safeguard them during storage. The overwrapped vials are cryogenically preserved in a facility that isclosely monitored at all times to ensure that your menstrual stem cells are safe and ready for future use.

What are the benefits of banking menstrual stem cells?Cryo-Cell's innovative menstrual stem cell banking service provides women with the exclusive opportunity to build their own personal healthcare portfolio with stem cells that will be a 100% match for the donor. Menstrual stem cells have demonstrated the capability of differentiating into many other types of stem cells such as cardiac, neural, bone, fat and cartilage.

Bankingmenstrual stem cells now is an investment in your future medical needs. Currently, they are being studied to treat stroke, heart disease, diabetes, neurodegenerative disease, and ischemic wounds in pre-clinical and clinical models.

Cryo-Cells activities for New York State residents are limited to collection, processing, and long-term storage ofmenstrual stem cells. Cryo-Cells possession of a New York State license for such collection, processing, and long-term storage does not indicate approval or endorsement of possible future uses or future suitability of these cells.

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Cryonics: does it offer humanity a chance to return from the …

The decision of a teenage girl to have her body cryogenically frozen in the hope of being reanimated by medical advances in the future is one with which many could sympathise. But does current evidence suggest the gamble will pay off, or does cryonics simply give desperate people false hope dressed up in the language of science?

There are two advances that make cryonics a little less far-fetched that it once was. The first is vitrification. As Arctic explorers and mountaineers have learned, humans are not designed to be frozen and defrosted. When our cells freeze, they fill with ice crystals, which break down cell walls as they expand, reducing our body to mush once it is warmed up again.

Vitrification prevents this by replacing the blood with a mixture of antifreeze-like chemicals and an organ preservation solution. When cooled to below -90C, the fluid becomes a glass-like solid.

The technique has substantially improved the reliability of freezing and thawing embryos, and particularly eggs, in fertility treatment and it works for small pieces of tissue and blood vessels. Earlier this year, scientists managed to cryogenically freeze the brain of a rabbit and recover it in an excellent state although it is not clear if the brains functions would have been preserved as well as its superficial appearance. However, even vitrifying larger structures, such as human kidneys for transplantation, has never been done clinically and remains some way off.

Barry Fuller, a professor in surgical science and low temperature medicine, at University College London, said: There is ongoing research into these scientific challenges, and a potential future demonstration of the ability to cryopreserve human organs for transplantation would be a major first step into proving the concept, but at the moment we cannot achieve that.

This is the growing appreciation that our personality, skills and memories are to some extent defined by the connections between neurons. This has led some to speculate that rather than bringing the actual body back to life, the brains contents could be downloaded on to a computer, allowing the person to live as a robot in the future.

This might have the whiff of nonsense, but Nick Bostrom, a professor of philosophy at the University of Oxfords Future of Humanity Institute, and his colleague, Anders Sandberg, are both banking on this possibility. As a head, my life would be limited, but by then we will be able to make real connections to computers, Anders said in a 2013 interview. So my hope is that, once revived, my memories and personality could be downloaded into a computer.

However, many neuroscientists have pointed out that even if you could code the astronomical number of connections between the brains 100bn neurons, even this would not capture the full complexity of the human mind.

From a purely scientific perspective, your money is probably better spent while you are still alive.

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Stem Cells For Heart Health: What The Current Research …

Stem cells are incredible. Science is only starting to scratch the surface of how these amazing cells can help people suffering from heart failure and other cardiovascular issues. Heres some information on what stem cells are, and how they may help heart attack patients and others who have problems involving their heart tissue.

There are more than 200 kinds of cells in the body, and each type is specifically structured for the job its supposed to do. There are skin cells, nerve cells, and cells that form heart tissue and other tissues in the body.1

Theyre found in bone marrow, blood vessels, the liver, the brain, and other parts of the body. Stem cells are even found in the umbilical cord. These sophisticated cells change over time as the body matures. Some of them disappear shortly after youre born, while others stay with you for a lifetime.2

There are three main types of stem cells tissue-specific (adult stem cells), embryonic stem cells, and induced pluripotent (iPS) stem cells. Heres a quick look at each type:

These typically reside in a specific organ, generating other cells to support the health of that organ. They replace those that are lost through injury, or through everyday living.3

Embryonic stem cells form about three to five days after a sperm fertilizes an egg. These are also known as pluripotent cells. This simply means they can develop into any sort of cell the body needs to develop.4

Embryonic cells have been the source of a massive controversy. The main reason is that harvesting these cells destroys the embryo.5 Scientists are working to develop iPS cells that come from adult stems cells rather than embryonic cells. Early research indicates that these cells may share many of the same characteristics of embryonic cells. But there are differences between the two, and there is more work to be done before scientists know exactly what those differences are.6

Research is ongoing into the potential use of stem cells for heart health. For example, work is being done to see if stem cells can help improve heart attack survival rates. Scientists are also looking into the potential for giving a patient their own cardiac stem cells after a heart attack, or even giving patients non-cardiac stem cells from a donor after an attack takes place.7

The goal of this research is to eventually provide cardiac patients with stem cells that can regenerate heart tissue that has been damaged. Some researchers feel that these advances are imminent, while others believe there is a great deal of work yet to be done.8

Early results from ongoing clinical trials involving stem cells for heart health are extremely promising. In one study, a group of 109 patients suffering from heart failure received either stem cell therapy or a placebo. According to the results, the patients who received stem cells were at significantly lower risk of hospitalization or death due to a sudden worsening of their condition.9

Heart failure affects more than 5 million people in the U.S.10 It occurs when the heart gradually weakens to the point to where it cant pump enough blood to meet the needs of the rest of the body. For those with severe heart failure, the only options are either to have a heart transplant or have a device planted to help the heart continue pumping. And even this is only a temporary measure theyll still need a transplant.11

Another study involved the use of stem cells from the umbilical cord. This trial involved 30 heart failure patients. Like the previous study, one group received stem cells while the other received a placebo. The umbilical cords were donated by healthy mothers whose babies were delivered through cesarean section.12

According to the results, the hearts of patients who received the umbilical cord stem cells pumped better than those of the placebo group. The stem cell patients also showed improved quality of life and day-to-day functioning. In addition, the stem cell group did not report any adverse effects, such as immune system reactions.13

As you can see, the use of stem cells to treat heart patients shows great promise. But this is still an extremely young scientific field, and a great deal more research must be performed. Many questions have to be answered, such as what approaches to stem cell harvesting will work the best and what types of side effects are possible from stem cell treatment.

However, this research does bring hope. And hope is something that is incredibly important to many of those suffering from severe cardiac illnesses.

Learn More:How Cardio Can Change Your Brain (And Why Thats Good News!)NEWS: A Vaccine For Arthritis Is Closer Than You ThinkAre Organ Donors At Risk of Becoming Obsolete?

Sources1.https://askabiologist.asu.edu/questions/human-cell-types2.https://www.medicalnewstoday.com/info/stem_cell3.http://www.closerlookatstemcells.org/learn-about-stem-cells/types-of-stem-cells4.https://stemcells.nih.gov/info/basics/3.htm5.http://www.cnn.com/2013/07/05/health/stem-cells-fast-facts/index.html6.http://www.closerlookatstemcells.org/learn-about-stem-cells/types-of-stem-cells#induced-pluripotent7.https://my.clevelandclinic.org/health/diseases/17508-stem-cell-therapy-for-heart-disease8.https://www.health.harvard.edu/heart-health/repairing-the-heart-with-stem-cells9.https://www.ncbi.nlm.nih.gov/pubmed/2705988710.https://www.cdc.gov/dhdsp/data_statistics/fact_sheets/fs_heart_failure.htm11.http://www.heart.org/HEARTORG/Conditions/HeartFailure/TreatmentOptionsForHeartFailure/Devices-and-Surgical-Procedures-to-Treat-Heart-Failure_UCM_306354_Article.jsp#.WleO-yMrJ3k12.https://www.medicalnewstoday.com/articles/319552.php13.http://circres.ahajournals.org/content/early/2017/09/15/CIRCRESAHA.117.310712

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Stem Cells For Heart Health: What The Current Research ...

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