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Researchers advance CRISPR-based tool for diagnosing disease …

The team that first unveiled the rapid, inexpensive, highly sensitive CRISPR-based diagnostic tool called SHERLOCK has greatly enhanced the tools power, and has developed a miniature paper test that allows results to be seen with the naked eye without the need for expensive equipment.

The SHERLOCK team developed a simple paper strip to display test results for a single genetic signature, borrowing from the visual cues common in pregnancy tests. After dipping the paper strip into a processed sample, a line appears, indicating whether the target molecule was detected or not.

This new feature helps pave the way for field use, such as during an outbreak. The team has also increased the sensitivity of SHERLOCK and added the capacity to accurately quantify the amount of target in a sample and test for multiple targets at once. All together, these advancements accelerate SHERLOCKs ability to quickly and precisely detect genetic signatures including pathogens and tumor DNA in samples.

Described today in Science, the innovations build on the teams earlier version of SHERLOCK (shorthand for Specific High Sensitivity Reporter unLOCKing) and add to a growing field of research that harnesses CRISPR systems for uses beyond gene editing. The work, led by researchers from the Broad Institute of MIT and Harvard and from MIT, has the potential for a transformative effect on research and global public health.

SHERLOCK provides an inexpensive, easy-to-use, and sensitive diagnostic method for detecting nucleic acid material and that can mean a virus, tumor DNA, and many other targets, said senior author Feng Zhang, a core institute member of the Broad Institute, an investigator at the McGovern Institute, and the James and Patricia Poitras 63 Professor in Neuroscience and associate professor in the departments of Brain and Cognitive Sciences and Biological Engineering at MIT. The SHERLOCK improvements now give us even more diagnostic information and put us closer to a tool that can be deployed in real-world applications.

The researchers previously showcased SHERLOCKs utility for a range of applications. In the new study, the team uses SHERLOCK to detect cell-free tumor DNA in blood samples from lung cancer patients and to detect synthetic Zika and Dengue virus simultaneously, in addition to other demonstrations.

Clear results on a paper strip

The new paper readout for SHERLOCK lets you see whether your target was present in the sample, without instrumentation, said co-first author Jonathan Gootenberg, a Harvard graduate student in Zhangs lab as well as the lab of Broad core institute member Aviv Regev. This moves us much closer to a field-ready diagnostic.

The team envisions a wide range of uses for SHERLOCK, thanks to its versatility in nucleic acid target detection. The technology demonstrates potential for many health care applications, including diagnosing infections in patients and detecting mutations that confer drug resistance or cause cancer, but it can also be used for industrial and agricultural applications where monitoring steps along the supply chain can reduce waste and improve safety, added Zhang.

At the core of SHERLOCKs success is a CRISPR-associated protein called Cas13, which can be programmed to bind to a specific piece of RNA. Cas13s target can be any genetic sequence, including viral genomes, genes that confer antibiotic resistance in bacteria, or mutations that cause cancer. In certain circumstances, once Cas13 locates and cuts its specified target, the enzyme goes into overdrive, indiscriminately cutting other RNA nearby. To create SHERLOCK, the team harnessed this off-target activity and turned it to their advantage, engineering the system to be compatible with both DNA and RNA.

SHERLOCKs diagnostic potential relies on additional strands of synthetic RNA that are used to create a signal after being cleaved. Cas13 will chop up this RNA after it hits its original target, releasing the signaling molecule, which results in a readout that indicates the presence or absence of the target.

Multiple targets and increased sensitivity

The SHERLOCK platform can now be adapted to test for multiple targets. SHERLOCK initially could only detect one nucleic acid sequence at a time, but now one analysis can give fluorescent signals for up to four different targets at once meaning less sample is required to run through diagnostic panels. For example, the new version of SHERLOCK can determine in a single reaction whether a sample contains Zika or dengue virus particles, which both cause similar symptoms in patients. The platform uses Cas13 and Cas12a (previously known as Cpf1) enzymes from different species of bacteria to generate the additional signals.

SHERLOCKs second iteration also uses an additional CRISPR-associated enzyme to amplify its detection signal, making the tool more sensitive than its predecessor. With the original SHERLOCK, we were detecting a single molecule in a microliter, but now we can achieve 100-fold greater sensitivity, explained co-first author Omar Abudayyeh, an MIT graduate student in Zhangs lab at Broad. Thats especially important for applications like detecting cell-free tumor DNA in blood samples, where the concentration of your target might be extremely low. This next generation of features help make SHERLOCK a more precise system.

The authors have made their reagents available to the academic community through Addgene and their software tools can be accessed via the Zhang lab website and GitHub.

This study was supported in part by the National Institutes of Health and the Defense Threat Reduction Agency.

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Researchers use CRISPR to detect HPV and Zika

The first study comes from the lab of CRISPR pioneer Jennifer Doudna. Her team discovered that a CRISPR system different from the CRISPR-Cas9 one we’re used to hearing about can not only snip away specific bits of double-stranded DNA, but can then also cut single-stranded DNA that’s near it. After they uncovered this ability of CRISPR-Cas12a, they used it to detect two common types of HPV. Once their CRISPR-Cas12a system detected HPV DNA in infected cells, it cleaved a another piece of DNA that then released a fluorescent signal, providing a visual sign of the presence of HPV. The researchers dubbed the system DETECTR and The Verge reports that it takes around an hour to work and costs less than a dollar.

The lab of another CRISPR pioneer, Feng Zhang, has now improved on a previous system it developed last year. SHERLOCK, as it’s called, can detect specific bits of DNA and RNA to determine whether viruses like Zika or dengue are present in a blood sample, identify mutations in tumor DNA and spot the presence of harmful bacteria. In their latest study, the research team describes SHERLOCK version 2.0, which is not only over three times as sensitive as the first version, but can also detect both Zika and dengue in the same sample. Their system uses several CRISPR enzymes, including Cas13 and Csm6, and can be loaded onto a paper strip, making it incredibly easy to use. You can see examples of the strips in the GIF below. Jonathan Gootenberg, one of the authors of the study, told The Verge, “The fact that we can put all these different enzymes into a single tube and have them not only play nice with each other, but also tell us information we couldn’t get otherwise — that is really spectacular and it speaks to a lot of the power of biochemistry.”

Lastly, Harvard University’s David Liu published a study showing that CRISPR can be used to track certain ongoings in a cell. Seeing what a cell has been exposed to in the past has been a rather hard thing to do, but CRISPR systems provide a way for researchers to do just that. Liu’s team used CRISPR in two different ways to record when a cell was exposed to certain chemicals. In the first, CRISPR was used to snip bits of DNA called plasmids if it came in contact with a particular chemical, such as an antibiotic or a nutrient. By comparing the ratio of the plasmid types that were destroyed by CRISPR to other, similar plasmids that were left alone, the researchers were able to determine just how often the cells were exposed to those chemicals. Another version of the system changed individual letters, or bases, of DNA rather than snipping plasmids and the team was able to determine when cells were exposed to antibiotics, nutrients, viruses and light by examining those changes in the DNA bases.

While all three of these systems need further development before they can be used outside of the lab, they show that CRISPR has quite a lot of uses, beyond just treating disease. The technology is incredibly versatile and we’re sure to see even more applications going forward.

Image: Zhang Lab, Broad Institute of MIT and Harvard

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Researchers use CRISPR to detect HPV and Zika

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Bone marrow | anatomy | Britannica.com

Bone marrow, also called myeloid tissue, soft, gelatinous tissue that fills the cavities of the bones. Bone marrow is either red or yellow, depending upon the preponderance of hematopoietic (red) or fatty (yellow) tissue. In humans the red bone marrow forms all of the blood cells with the exception of the lymphocytes, which are produced in the marrow and reach their mature form in the lymphoid organs. Red bone marrow also contributes, along with the liver and spleen, to the destruction of old red blood cells. Yellow bone marrow serves primarily as a storehouse for fats but may be converted to red marrow under certain conditions, such as severe blood loss or fever. At birth and until about the age of seven, all human marrow is red, as the need for new blood formation is high. Thereafter, fat tissue gradually replaces the red marrow, which in adults is found only in the vertebrae, hips, breastbone, ribs, and skull and at the ends of the long bones of the arm and leg; other cancellous, or spongy, bones and the central cavities of the long bones are filled with yellow marrow.

Red marrow consists of a delicate, highly vascular fibrous tissue containing stem cells, which differentiate into various blood cells. Stem cells first become precursors, or blast cells, of various kinds; normoblasts give rise to the red blood cells (erythrocytes), and myeloblasts become the granulocytes, a type of white blood cell (leukocyte). Platelets, small blood cell fragments involved in clotting, form from giant marrow cells called megakaryocytes. The new blood cells are released into the sinusoids, large thin-walled vessels that drain into the veins of the bone. In mammals, blood formation in adults takes place predominantly in the marrow. In lower vertebrates a number of other tissues may also produce blood cells, including the liver and the spleen.

Because the white blood cells produced in the bone marrow are involved in the bodys immune defenses, marrow transplants have been used to treat certain types of immune deficiency and hematological disorders, especially leukemia. The sensitivity of marrow to damage by radiation therapy and some anticancer drugs accounts for the tendency of these treatments to impair immunity and blood production.

Examination of the bone marrow is helpful in diagnosing certain diseases, especially those related to blood and blood-forming organs, because it provides information on iron stores and blood production. Bone marrow aspiration, the direct removal of a small amount (about 1 ml) of bone marrow, is accomplished by suction through a hollow needle. The needle is usually inserted into the hip or sternum (breastbone) in adults and into the upper part of the tibia (the larger bone of the lower leg) in children. The necessity for a bone marrow aspiration is ordinarily based on previous blood studies and is particularly useful in providing information on various stages of immature blood cells. Disorders in which bone marrow examination is of special diagnostic value include leukemia, multiple myeloma, Gaucher disease, unusual cases of anemia, and other hematological diseases.

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Bone marrow | anatomy | Britannica.com

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Hypogonadism: Practice Essentials, Background, Pathophysiology

Morbidity for men and women with hypogonadism includes infertility and an increased risk of osteoporosis; there is no increase in mortality.

Hypogonadotropic hypogonadism (see the image below) is one of several types of hypogonadism.

History

Considerations in the evaluation of males with hypogonadism include the following:

For postpubertal males, the rate of beard growth, libido and sexual function, muscle strength, and energy levels

Possible causes of acquired testicular failure (eg, mumps orchitis, trauma, radiation exposure of the head or testes, and chemotherapy)

Drugs that may interrupt testicular function -Including agents that interfere with testosterone synthesis, such as spironolactone andcyproterone.Agents such as cortisol,marijuana, heroin, and methadone may interfere with gonadotropin secretion.

Considerations in the evaluation of females with hypogonadism include the following:

Signs associated with Turner syndrome (eg, lymphedema, cardiac or renal congenital anomalies, and short growth pattern)

Age of menarche

Physical examination

Considerations in the physical examination of males with hypogonadism include the following:

Evaluation of the testes: This is the most important feature of the physical examination; determine whether both testes are palpable, their position in the scrotum, and their consistency; testes size can be quantitated by comparison with testicular models (orchidometer), or their length and width may be measured

Examination of the genitalia for hypospadias

Examination of the scrotum to see if it is completely fused

Evaluation of the extent of virilization

Staging of puberty: Use the Tanner criteria for genitalia, pubic hair, and axillary hair

Examination for signs of Klinefelter syndrome (eg, tall stature, especially if the legs are disproportionately long, gynecomastia, small or soft testes, and a eunuchoid body habitus)

Considerations in the physical examination of females with hypogonadism include the following:

Examination of the genitalia is important

Determination of the extent of androgenization: May be adrenal or ovarian in origin and is demonstrated in pubic and axillary hair

Determination of the extent of estrogenization: As evidenced by breast development and maturation of the vaginal mucosa

Examination for signs of Turner syndrome (eg, short stature, webbing of the neck [such as pterygium colli], a highly arched palate, short fourth metacarpals, widely spaced nipples, or multiple pigmented nevi)

See Clinical Presentation for more detail.

The following studies may be indicated in males with hypogonadism:

Follicle-stimulating hormone (FSH) levels

Luteinizing hormone (LH) levels

Prolactin levels

Testosterone levels

Thyroid function

Seminal fluid examination

Karyotyping

Testicular biopsy

For males after puberty, the Guidelines of the Endocrine Society [2] require that the diagnosis of hypogonadism be based on symptoms and signs of hypogonadism plus the presence of a low testosterone level measured on at least 2 occasions.

The following studies may be indicated in females with hypogonadism:

Additional tests in the evaluation of patients with hypogonadism include the following:

Adrenocorticotropic hormone (ACTH) stimulation testing: In patients in whom a form of congenital adrenal hyperplasia is suspected, adrenal steroid synthesis is best evaluated by performing a cosyntropin (ACTH 1-24) stimulation test

Luteinizing-hormone releasing hormone (LHRH) stimulation testing: To distinguish between true hypogonadotropic hypogonadism and constitutional delay in growth and maturation

Testicular tissue testing: If the testes are not palpable and if it is not certain whether any testicular tissue is present, administering human chorionic gonadotropin (hCG) and measuring testosterone response may be helpful

See Workup for more detail.

Hormonal replacement

The simplest and most successful treatment for males and females with either hypergonadotropic or hypogonadotropic hypogonadism is replacement of sex steroids, but the therapy does not confer fertility or, in men, stimulate testicular growth.

When fertility is desired, an alternative therapy for men with hypogonadotropic hypogonadism is administration of pulsatile LHRH or injections of hCG and FSH. (In patients with hypergonadotropic hypogonadism, fertility is not possible.)

In a 6-year European study of men being treated for hypogonadism, long-term transdermal testosterone treatment did not increase prostate-specific antigen (PSA) levels or influence prostate cancer risk. [3, 4]

Investigators used data from a 5-year, open-label extension of a 1-year trial of a transdermal testosterone patch (Testopatch) in men with hypogonadism. Study subjects wore two 60 cm2 patches, each of which delivered 2.4 mg of testosterone per day. More than 90% of patients had PSA concentrations below 2 ng/mL during the 6-year study, and no prostate cancer was found in patients over the course of the trial.

See Treatment and Medication for more detail.

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Home – STEM CELL SCIENCE

Stem cell can be isolated from the the bone marrow and adipose tissue in the abdomen that are capable of forming new blood vessels and heart muscle cells. The cell number is so small in the tissues that the cells should be grown for several weeks before there is enough for the treatment of patients.

We have conducted three clinical stem cell therapy studies in which patients with coronary artery disease havebeen treated with their own mesenchymal stem cells from either the bone marrow or adipose tissue. Encouraging results are available from two studies and there is ongoing follow-up in the third study. Treatments with stem cells have in all previous studies been without any side effects.

During the course of the SCIENCE study a total of 138 patients with heart failure will be included and treated in a so-called blinded placebo-controlled design. This means that 92 patients will receive stem cells and 46 patients placebo (inactive medication, saline). Choice of treatment will be done by drawing lots. The study is carried out by an international collaboration between cardiac centers in Denmark, Poland, Germany, Netherlands, Austria and Sloveniaand the industrial partners Terumo BCT and COOK Tegentec.

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Home – STEM CELL SCIENCE

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Slideshow: Hormone Imbalance: Symptoms and Treatment

IMAGES PROVIDED BY:

1) Getty Images

2) Amie Brink/WebMD

3) Thinkstock Photos

4) Thinkstock Photos

5) Getty Images

6) Thinkstock Photos

7) Thinkstock Photos

8) Getty Images

9) Getty Images

10) Getty Images

11) Thinkstock Photos

12) Thinkstock Photos

13) Photolibrary.com

SOURCES:

David Adamson, M.D., clinical professor, Stanford University School of Medicine, CEO of ARC Fertility, Saratoga, California.

Alyssa Dweck, M.D., assistant clinical professor of obstetrics and gynecology, Mount Sinai School of Medicine, New York City.

Jenna LoGiudice, PhD, CNM, RN, assistant professor, Fairfield Universitys School of Nursing, Fairfield, CT.

American Academy of Dermatology: Hormonal Factors Key to Understanding Acne in Women

Cleveland Clinic: Menstrual Cycle

Gao, Q., Endocrinology and Metabolism, May 2008

Gov.UK: Hormone Headaches

Harvard Medical School: Testosterone Therapy: Is It For Women? Perimenopause: Rocky road to menopause, Dealing With Menopause Symptoms

Johns Hopkins Medicine: Hormone Imbalance May Be Causing Your Acne

Lopez, M., Trends in Molecular Medicine, July 2013

National Cancer Institute: Understanding Breast Changes

National Sleep Foundation: Menopause and Sleep

Soares, C. Journal of Psychiatry and Neuroscience, July 2008

The University of Connecticut Health Center: Benign Diseases of the Breast

The University of North Carolina School of Medicine: Hormones and IBS

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Slideshow: Hormone Imbalance: Symptoms and Treatment

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LABOKLIN (UK)| Genetic Diseases | Dogs| Dwarfism …

Dwarfism (Pituitary Dwarfism / Hypopituitarism)

Test number: 8142

DWARFISM

clear

100% clear

clear

carrier

50% clear + 50% carriers

clear

affected

100% carriers

carrier

clear

50% clear + 50% carriers

carrier

carrier

25% clear + 25% affected + 50% carriers

carrier

affected

50% carriers + 50% affected

affected

clear

100% carriers

affected

carrier

50% carriers + 50% affected

affected

affected

100% affected

Clear

Genotype: N / N [ Homozygous normal ]

The dog is noncarrier of the mutant gene.

Carrier

Genotype: N / DWARFISM [ Heterozygous ]

The dog carries one copy of the mutant gene and one copy of the normal gene.

Carriers should only be bred to clear dogs.

Avoid breeding carrier to carrier because 25% of their offspring is expected to be affected (see table above)

Affected

Genotype: DWARFISM / DWARFISM [ Homozygous mutant ]

The dog carries two copies of the mutant gene and therefore it will pass the mutant gene to its entire offspring.

By DNA testing, the responsible mutation can be shown directly. This method provides a test with a very high accuracy. It offers the possibility to distinguish not only between affected and clear dogs, but also to identify clinically healthy carriers. This is an essential information for controlling the condition in the breed, as carriers are able to spread the disease in the population.

test will be performed at a partner laboratory

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LABOKLIN (UK)| Genetic Diseases | Dogs| Dwarfism …

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CRISPR Gene Editing And 3 Biotech Companies Blaze New Path To …

Imagine editing one gene and curing a debilitating disease.Three small biotech companies with combined annual sales of less than $50 million Crispr Therapeutics (CRSP), Intellia Therapeutics (NTLA) and Editas Medicine (EDIT) say that soon could be a reality.

X All three biotech stocks went public in 2016 to bet big on a simple premise: Altering specific genes can create curative medicines.An estimated 5,000 diseases could be cured by changing one targeted gene, says former Intellia Chief Executive Nessan Bermingham.

The World Health Organization has a higher estimate for what are known as monogenic diseases and says it’s actually north of 10,000.

“People have been talking about (personalized medicine) for 20 years and yet we’ve never had a system to allow us to do it before,” Bermingham told Investor’s Business Daily before stepping down from his role on Dec. 31.”And for the first time ever, we actually have a system to do it and that system would be based on your personalized genome.”

That system is known as CRISPR, and it’s where Crispr, Intellia and Editas are putting their chips. It’s a cheaper and faster gene editing method and, according to Bermingham, the key to advancing personalized medicine. Some analysts think CRISPR technology could provide the platform for the next generation of giant biotech companies.

CRISPR the technology not to be confused with Crispr Therapeutics, the company builds on a project that sequenced the human genome. The first map cost $2.7 billion and was completed in 2003.

Since then, the cost to map an individual’s genome has dropped precipitously and could come down to just hundreds of dollars in the next few years, Bermingham says. Large-data analytics also have a part to play in sifting through the genome.

IBD’S TAKE:Biotech companies account for a large share of recent IPO stocks, yet investing in them before they have profits or sales can be risky. Learn to identify the best IPOs and how to trade themfor potential big gains.

When the first human genome was mapped, investigators were “absolutely horrified” to find just 20,000 genes in the human body that code proteins, Bermingham says. That was down from estimates of 100,000. Essentially, these protein-coding genes serve as words in the genetic language.

Investigators also found regions of DNA that were initially thought to have no purpose. These were controversially called “junk DNA” that does not code protein. But, as it turns out, these sequences do have a key purpose in regulating the expression of genes.

All together, the better understanding of the human genome has allowed these biotech companies to utilize CRISPR, an acronym for the technology known asClustered Regularly Interspaced Short Palindromic Repeats.

There is a caveat, however. In January, a paper published by bioRxiv said there may be evidence that human immune systems may fight off the major form of genome editing that uses an enzyme called Cas9, thus rendering the science ineffective. The paper, however, has yet to be peer reviewed.

The process, developed at various universities, essentially uses specialized strands of DNA thatact as molecular “scissors.” Those scissors are capable of editing other DNA at specific points, and allow biotech companies to edit, add or remove faulty genes responsible for diseases.

There are varying types of scissors. Crispr, Intellia and Editas are using the Cas9 CRISPR technology, ARK Invest analyst Manisha Samy told IBD. She estimates Cas9 can reach 70%-80% of the human genome. Developing new scissors can expand the reach into more genes and diseases, she says.

Gene editing isn’t new, she adds. Older techniques called TALENs and zinc finger nucleases have been around for some time. Notably, biotech companyBluebird Bio (BLUE) is using a variation of TALENs, and Sangamo Therapeutics (SGMO) is using a method of zinc finger nucleases.

She likens CRISPR technology to a word processor.

“We think CRISPR gene editing is analogous to a DNA word processor with two functions: find and delete,” she said in a January 2017 report. “In addition, scientists are working on a rudimentary paste function, allowing CRISPR to insert appropriate DNA code to repair mutations.”

Older technologies used by biotech companies are more like old-fashioned typewriters, requiring actual cutting and pasting, she says. CRISPR technology is also cheaper and easier to use than TALENs and zinc fingers, says JMP Securities analyst Mike King.

“What’s so powerful about CRISPR is it’s so easy to use,” he told IBD. “High school students are doing experiments in the biology lab to knock out genes. Zinc fingers takes a lot of talent and time. You have to fiddle with them a lot. The systems created under CRISPR are quite robust.”

In January, bioRxiv an online archive and distribution service for unpublished reports in the life sciences field published a paper casting doubt on the durability of CRISPR gene therapy over time, suggesting the body could build an immunity to it. Analysts and biotech companies are not worried, however, saying either this is a nonissue or there’s time for the science to catch up.

Many companies working in CRISPR are doing so using the Cas9 enzyme, short for CRISPR associated protein 9. Cas9 is derived from two bacteria that cause infections in humans at high rates, meaning some immune systems could have developed immunities to them.

Would CRISPR gene editing, using that enzyme, work in those patients?

It depends, Crispr Therapeutics said in a follow-up email to IBD. It’s important to note the lead investigator and writer on the bioRxiv paper wasMatthew Porteus, a scientific founder and advisory board member for Crispr Therapeutics.

When the gene editing is done ex vivo, or outside the body, the Cas9 enzyme is degraded and, therefore, essentially gone by the time the cells are reintroduced to the patient, Crispr told IBD.

For in vivo applications, when gene editing is done inside the body, Crispr Therapeutics says it uses several approaches to ensure transient expression of the Cas9 enzyme. Because of that, “we do not expect pre-existing immunity to Cas9 to cause any issues,” the firm said.

Ark’s Samy also noted that other enzymes are in use. Editas is also using the Cpf1 enzyme. This enzyme is derived from other bacteria and could overcome some of the immunity challenges involving Cas9.

Intellia told IBD in a follow-up email that in clinical testing, its delivery system for treatment in rodents and non-human primates has yet to falter. Further, Intellia notes it’s using an advanced form of Cas9 and none of the donors had a pre-existing immunity in its study.

The data are still early. Editas has done its own work in immune responses to CRISPR genome editing and will present a paper in the future, JMP’s King said in a Jan. 8 note to clients. Management has indicated it found immune responses to be “much lower” than those reported in the other paper.

“Immune responses are not uncommon,” Samy said. “Scientists have worked for decades on evading immune recognition. There are numerous workarounds that can be implemented to reduce any potential side effects with Cas9 and we have proved this in a number of other therapeutic modalities.”

Among the biotech companies, Crispr Therapeutics is ahead of the competition from a regulatory standpoint. On Dec. 7, the firm submitted its first application for a clinical trial testing its gene therapy, known as CTX001, in a blood disorder known as beta thalassemia.

The company is working with Vertex Pharmaceuticals (VRTX) in beta thalassemia, as well as sickle cell disease. The therapies are part of Crispr’s ex vivo programs, where gene editing is done on cells outside the body before they are reintroduced to the patient. Crispr is also looking at in vivo therapies for the liver, muscles and lungs.

According to a Crispr news release, the trial is set to begin in Europe in 2018 in adult patients. This is expected to be the first in-human trial of a gene editing treatment based on CRISPR technology. Crispr also plans to file an application to begin testing for CTX001 in treating sickle cell disease in the U.S. in 2018.

Intellia also has in vivo and ex vivo programs in gene editing, and also is working in sickle cell disease. It’s furthest along in a partnership with Regeneron Pharmaceuticals (REGN) for a therapy to treat what’s known as transthyretin amyloidosis, a condition characterized by the buildup of abnormal protein deposits throughout the body.

Alnylam Pharmaceuticals (ALNY) and Ionis Pharmaceuticals (IONS) also are working separately to treat the disease using different methods called RNA interference and antisense technology, respectively.

Meanwhile, Editas is working on an injected treatment for an inherited eye disease known as Leber congenital amaurosis, which is characterized by severe loss of vision at birth. It is also using gene editing in sickle cell disease and beta thalassemia.

Intellia and Editas also are expected to start in-human trials in 2018, analysts say, though Intellia has not said when it will begin testing.

Regulators are getting more comfortable with the idea of gene editing, Crispr Therapeutics President Sam Kulkarni told IBD. The benefit of gene editing and potential trouble with it is that it’s meant to be a permanent fix. The biotech companies are working to ensure they hit a bull’s-eye every time out.

“We’ve shown you we can make this edit and it’s done in a precise fashion using (targets the industry calls) molecular ZIP codes,” he said. “We eliminate edits happening outside places you want them to happen. And we manufacture these in a high-quality fashion, understanding the pharmacology.”

Both Kulkarni and Intellia’s Bermingham who was succeeded byJohn Leonard, a former AbbVie (ABBV) executive say there’s room for all three big players in the group.

Sizing the market is a challenge, ARK’s Samy says. No matter how you slice it, the numbers are big and a lot will depend on which diseases companies target and how they set pricing.

If CRISPR is able to address all monogenic diseases diagnosed each year, that’s a $75 billion market globally, she says. Addressing all these diseases for people already living with diagnoses would be a $2 trillion market.

“One product is not going to cure everything,” she said. “Whenever you’re seeing volatility between these three main CRISPR companies, it doesn’t really make sense because there’s room for all of them and more when it comes to CRISPR.”

Kulkarni says it’s unlikely the market will remain at just three publicly traded biotech companies with CRISPR technology in the long run. The technology is just that remarkable.

“Once in a lifetime may be a little bit of a stretch, maybe not,” he said. “But it’s definitely a once in a generation type of advance in the field. The last time this kind of excitement happened in the biotech field was when antibodies were applied as therapeutic modalities. On the basis of that, technology companies like Genentech (now owned byRoche (RHHBY)) were created.”

He added: “Here we have the basis of a CRISPR platform to create the next big biotech giants.”

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CRISPR Gene Editing And 3 Biotech Companies Blaze New Path To …

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Prevalence of Hypopituitarism in Veterans 42% A NEW …

Why are they not doing something with this information? At least 2 million of our Veterans are needlessly suffering when treatment is available!!

Research showsabout 24% of US Veterans who return home from war suffer from PTSD. (With21.8 million veterans of the U.S. armed forces as of 2014, that means about 5.2 million Veterans suffer from PTSD.)Research shows42% of those Veterans who come home with PTSD actually have Hypopituitarism, and when treated, their PTSD symptoms (including depression, and other mental and physical health disorders) actually go away!

That means atleast 2 million Veterans in the United States are needlessly suffering from undiagnosed hypopituitarism. And of Veterans diagnosed with a Traumatic Brain Injury from war, the percent who may have hypopituitarism could be as high as 80%.

What is hypopitutarism? Its when the brain is not able to send signals to cells throughout the body to control all things homeostasis. Everything metabolic. Everything that makes you human. Blood, heart, bone, and muscle function, mental health, sleep cycle, reproductive function, ability to heal and fight infection, and much more. Without these brain signals, you are always unwell, and sentenced to a life of illness and certainlyan early death.

When you google Veterans and PTSD, about 25millionresults come back. When you google Veterans and Hypopituitarism, only 226,000 results come back. Yet nearly half of Veterans with PTSD actually have hypopituitarism. This awareness should spread like wildfire, 2 million veterans may get their lives back.

So why arent they getting a diagnosis? Because doctors dont know to lookfor the symptoms and they dont know the proper tests. The only doctors who are taught about Hypopit are endocrinologists, and they are taught that it is rare. They are misinformed by their textbooks and, admittedly, due to lack of research, there is gross missing information. Hypopit patients find medical professionals actually know very little about diagnosis, testing and treatment. Often times, Hypopit patients are put on anti-anxiety pills and antidepressants, instead of the treatment they need. A bandaid doesnt fix a bullet hole, it may cover it up for a little while, but the problem still exists. We need the textbooks to teach doctors that Hypopit is not rare and we need to teach them that anyone who has symptoms and has experienced a traumatic event should be properly tested.

2010 A recommendation was made by AMSUS (the Society of the Federal Health Professional) for hormonal testing of veterans who sustained and sort of traumatic brain injury.

Military Medicine Recent civilian data obtained in those sustaining head injuries, has found a high prevalence of pituitary dysfunction. Currently, there is no data available in the military population. We reviewed the literature for traumatic brain injury (TBI)-related hypopituitarism and found that the prevalence of anterior hypopituitarism may be as high as 3080% after 2436 months. Since many of the symptoms of hypopituitarism are similar to those of TBI, it is important to make clinicians caring for combat veterans aware of its occurrence. Herein, we provide an overview of the literature and recommendations for hormonal testing when TBI-related hypopituitarism is suspected.Read the full article here:

http://publications.amsus.org/doi/abs/10.7205/MILMED-D-09-00189

2013- Science Daily reported, Up to 20 percent of veterans returning from Afghanistan and Iraq have experienced at least one blast concussion. New research suggests that nearly half these veterans may have a problem so under-recognized that even military physicians may fail to look for it. A new study conducted by Charles W. Wilkinson, Elizabeth A. Colasurdo, Kathleen F. Pagulayan, Jane. B. Shofer, and Elaine R. Peskind, all of the VA Puget Sound Health Care System and the University of Washington in Seattle, has found that about 42 percent of screened veterans with blast injuries have irregular hormone levels indicative of hypopituitarism.View the article here:

http://www.sciencedaily.com/releases/2013/04/130422102029.htm

2013 American Physiological Society (APS). Nearly half of U.S. veterans found with blast concussions might have hormone deficiencies. .

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Naturopathy NJ – Dr. Jen Phillips

Merchantville Office41 West Chestnut Ave.Merchantville, NJ 08109Call (856) 488-7067 for appointments

Moorestown OfficeMoorestown Office Center110 Marter AvenueSuite 408Moorestown, NJ 08057

Sewell Office – New LocationAgeless Skin and Laser Center660 Woodbury-Glassboro RdSewell, NJ 08080-2664Timberline Shopping CenterOffice is located behind Dunkin Donuts

Jennifer Phillips, ND

Dr. Jennifer Phillips is a board certified and licensed Naturopathic Physician.

She holds a BS in Biology and earned her Doctorate in Naturopathic Medicine from Bastyr University and Completed a residency specializing in adjuvant cancer therapy under the guidance of John Catanzaro, ND, author of Cancer, An Integrative Approach.

In her practice the emotional aspects of health and disease are explored in conjunction with lifestyle and nutritional management.

The Moorestown office is now located on Marter avenue across from the TD Bank. It is very close to the Big Acme in Moorestown that closed, and also near Wegmans.

I now offer a Wellness Package, and my rates for additional appointments have been updated.

Read about my rate increase in the Frequently Asked Questions section of Naturopathy NJ.

Read testimonials of those who have experienced better health through Natural Medicine.

Attention Women: Are you interested in an alternative to Mammography for Breast Cancer Detection?

Education Not Medication – click here for the truth about breast cancer

Jennifer Phillips, ND Merchantville Office (856) 488-7067 Merchantville Office 41 W Chestnut Ave, Merchantville, NJ 08109 US Moorestown Office 110 Marter Avenue Suite 408, Moorestown, NJ 08057 US Sewell Office (Ageless Skin and Laser Center) 660 Woodbury-Glassboro Rd, Sewell, NJ 08080-2664 US DrJen@naturopathynj.com

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Gene therapy | medicine | Britannica.com

Gene therapy, also called gene transfer therapy, introduction of a normal gene into an individuals genome in order to repair a mutation that causes a genetic disease. When a normal gene is inserted into the nucleus of a mutant cell, the gene most likely will integrate into a chromosomal site different from the defective allele; although that may repair the mutation, a new mutation may result if the normal gene integrates into another functional gene. If the normal gene replaces the mutant allele, there is a chance that the transformed cells will proliferate and produce enough normal gene product for the entire body to be restored to the undiseased phenotype.

Human gene therapy has been attempted on somatic (body) cells for diseases such as cystic fibrosis, adenosine deaminase deficiency, familial hypercholesterolemia, cancer, and severe combined immunodeficiency (SCID) syndrome. Somatic cells cured by gene therapy may reverse the symptoms of disease in the treated individual, but the modification is not passed on to the next generation. Germline gene therapy aims to place corrected cells inside the germ line (e.g., cells of the ovary or testis). If that is achieved, those cells will undergo meiosis and provide a normal gametic contribution to the next generation. Germline gene therapy has been achieved experimentally in animals but not in humans.

Scientists have also explored the possibility of combining gene therapy with stem cell therapy. In a preliminary test of that approach, scientists collected skin cells from a patient with alpha-1 antitrypsin deficiency (an inherited disorder associated with certain types of lung and liver disease), reprogrammed the cells into stem cells, corrected the causative gene mutation, and then stimulated the cells to mature into liver cells. The reprogrammed, genetically corrected cells functioned normally.

Prerequisites for gene therapy include finding the best delivery system (often a virus, typically referred to as a viral vector) for the gene, demonstrating that the transferred gene can express itself in the host cell, and establishing that the procedure is safe. Few clinical trials of gene therapy in humans have satisfied all those conditions, often because the delivery system fails to reach cells or the genes are not expressed by cells. Improved gene therapy systems are being developed by using nanotechnology. A promising application of that research involves packaging genes into nanoparticles that are targeted to cancer cells, thereby killing cancer cells specifically and leaving healthy cells unharmed.

Some aspects of gene therapy, including genetic manipulation and selection, research on embryonic tissue, and experimentation on human subjects, have aroused ethical controversy and safety concerns. Some objections to gene therapy are based on the view that humans should not play God and interfere in the natural order. On the other hand, others have argued that genetic engineering may be justified where it is consistent with the purposes of God as creator. Some critics are particularly concerned about the safety of germline gene therapy, because any harm caused by such treatment could be passed to successive generations. Benefits, however, would also be passed on indefinitely. There also has been concern that the use of somatic gene therapy may affect germ cells.

Although the successful use of somatic gene therapy has been reported, clinical trials have revealed risks. In 1999 American teenager Jesse Gelsinger died after having taken part in a gene therapy trial. In 2000 researchers in France announced that they had successfully used gene therapy to treat infants who suffered from X-linked SCID (XSCID; an inherited disorder that affects males). The researchers treated 11 patients, two of whom later developed a leukemia-like illness. Those outcomes highlight the difficulties foreseen in the use of viral vectors in somatic gene therapy. Although the viruses that are used as vectors are disabled so that they cannot replicate, patients may suffer an immune response.

Another concern associated with gene therapy is that it represents a form of eugenics, which aims to improve future generations through the selection of desired traits. Some have argued that gene therapy is eugenic but that it is a treatment that can be adopted to avoid disability. To others, such a view of gene therapy legitimates the so-called medical model of disability (in which disability is seen as an individual problem to be fixed with medicine) and raises peoples hopes for new treatments that may never materialize.

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Autologous cardiac-derived cells for advanced ischemic …

Disease Team Award DR1-01461, autologous cardiac-derived cells for advanced ischemic cardiomyopathy, is targeted at developing novel therapies for the treatment of heart failure, a condition which afflicts 7 million Americans. Heart failure, when symptomatic, has a mortality exceeding that of many malignant tumors; new therapies are desperately needed. In the second year of CIRM support, pivotal pre-clinical studies have been completed. We have found that dose-optimized injection of CSps preserves systolic function, attenuates remodeling, decreases scar size and increases viable myocardium in a porcine model of ischemic cardiomyopathy. The 3D microtissues engraft efficiently in preclinical models of heart failure, as expected from prior work indicating their complex multi-layer nature combining cardiac progenitors, supporting cells and derivatives into the cardiomyocyte and endothelial lineages. Analysis of the MRI data continues. We have developed standard operating procedures for cardiosphere manufacturing and release criteria, product and freezing/thawing stability testing have been completed for the 3D microtissue development candidate. We have identified two candidate potency assays for future development. The disease team will evaluate the results of the safety study (immunology, histology, and markers of ischemic injury) and complete the pivotal pig study in Q1 2012. With data in hand, full efforts will be placed on preparation of the IND for Q2 2012 submission.

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Autologous cardiac-derived cells for advanced ischemic …

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Hypogonadism – UCSF Medical Center

Hypogonadism is a condition that causes decreased function of the gonads, which are the testis in males and the ovaries in females, and the production of hormones that play a role in sexual development during puberty. You may be born with the condition or it can develop later in life from injury or infection. Some types of hypogonadism can be treated with hormone replacement therapy.

There are two forms of the condition primary hypogonadism resulting from problems of the testis or ovary and central hypogonadism caused by problems with the pituitary or hypothalamic glands. Central hypogonadism leads to decreased levels of luteinizing hormone (LH) and follicle stimulating hormones (FSH), released by the pituitary gland.

The condition may have genetic, menopausal autoimmune and viral causes or may develop after cancer treatments such as radiation and chemotherapy.

Fasting, weight loss, eating disorders such as anorexia nervosa, and bulimia, and stressful conditions can cause the condition.

In children before puberty, hypogonadism causes no symptoms. In adolescents, it can delay or prevent exual development.

Adult women with the condition may stop menstruating or develop infertility, loss of libido, vaginal dryness and hot flashes. Prolonged periods of hypogonadism can cause osteoporosis.

Men with the condition may experience loss of libido, erectile dysfunction and infertility.

To diagnose hypogonadism, tests may be performed to check hormone levels estogren in females and testosterone in males. In addition, levels of luteinizing hormone (LH) and follicle stimulating hormones (FSH) will be tested. LH and FSH are pituitary hormones that are stimulated by the gonads.

Other tests may measure thyroid hormones, sperm count and prolactin, a hormone released by the pituitary gland that stimulates breast development and milk production Tests also may be performed to test for anemia and possible genetic causes of symptoms.

For women, your doctor may request a sonogram of your ovaries.

If pituitary disease is suspected, a magnetic resonance imaging (MRI) scan or computed tomography (CT) scan may be performed to examine the the pituitary gland.

Hormone replacement therapy has proven to be effective treatment for hypogonadism in men and pre-menopausal women.

Estrogen may be administered in the form of a patch or pill. Testosterone can be given by a patch, a product soaked in by the gums, a gel or by injection.

For women who have not had their uterus removed, a combination of estrogen and progesterone is often recommended to decrease the chance of developing endometrial cancer. Low-dose testosterone may be added for women with hypogonadism who have a low sex drive.

Other hormones may be prescribed to restore fertility in men and women.

Reviewed by health care specialists at UCSF Medical Center.

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Hypogonadism – UCSF Medical Center

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Hairy skin from mouse stem cells may hold a cure for …

In a finding that may provide a potential cure for baldness, researchers have used stem cells from mice to develop a skin patch that is complete with hair follicles in a laboratory.

Using the skin model, the scientists developed both the epidermis (upper) and dermis (lower) layers of skin, which grow together in a process that allows hair follicles to form the same way as they would in a mouses body.

The novel skin tissue more closely resembles natural hair than existing models and may prove useful for testing drugs, understanding hair growth, and reducing the practice of animal testing, the researchers said.

You can see the organoids with your naked eye, said Karl Koehler, assistant professor at the Indiana University. It looks like a little ball of pocket lint that floats around in the culture medium. The skin develops as a spherical cyst, and then the hair follicles grow outward in all directions, like dandelion seeds.

The scientists developed both the epidermis (upper) and dermis (lower) layers of skin, which grow together in a process that allows hair follicles to form the same way as they would in a mouses body.(Getty Images/iStockphoto)

In the study, published in Cell Reports, Koehler and team originally began using pluripotent stem cells from mice, which can develop into any type of cells in the body, to create organoids — miniature organs in vitro — that model the inner ear.

But they discovered that they were generating skin cells in addition to inner ear tissue. Thus, they decided to coax the cells into sprouting hair follicles. Moreover, they found that mouse skin organoid technique could be used as a blueprint to generate human skin organoids.

It could be potentially a superior model for testing drugs, or looking at things like the development of skin cancers, within an environment thats more representative of the in vivo microenvironment, Koehler noted.

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Bone Marrow Transplantation | Bone Marrow Transplant …

Bone marrow is the spongy tissue inside some of your bones, such as your hip and thigh bones. It contains immature cells, called stem cells. The stem cells can develop into red blood cells, which carry oxygen throughout the body, white blood cells, which fight infections, and platelets, which help the blood to clot.

A bone marrow transplant is a procedure that replaces a person’s faulty bone marrow stem cells. Doctors use these transplants to treat people with certain diseases, such as

Before you have a transplant, you need to get high doses of chemotherapy and possibly radiation. This destroys the faulty stem cells in your bone marrow. It also suppresses your body’s immune system so that it won’t attack the new stem cells after the transplant.

In some cases, you can donate your own bone marrow stem cells in advance. The cells are saved and then used later on. Or you can get cells from a donor. The donor might be a family member or unrelated person.

Bone marrow transplantation has serious risks. Some complications can be life-threatening. But for some people, it is the best hope for a cure or a longer life.

NIH: National Heart, Lung, and Blood Institute

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Bone Marrow Transplantation | Bone Marrow Transplant …

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Hormone Replacement Clinic NY, Testosterone Injections for …

You may be reading this because you are not your normal self. Youre lethargic and your energy level is not what it was. Your body is becoming soft and flabby and youre having problems with focus and concentration. Your sex drive is down and you may be having difficulties achieving an erection. Low Testosterone or a Low T count may be responsible, and Hormone Replacement Therapy may be right for you.

According to the US Food and Drug Administration (FDA), 4 to 5 million American men may suffer from low testosterone, but only 5% are currently treated. What about the remaining 95%? Could you be one of them?

If you answered yes to more than half of these questions, chances are your testosterone levels are less than optimal and you may be deficient and benefit from Hormone Replacement Therapy. You may be going through the male menopause, a condition known as andropause.

Andropause refers to a set of gradual physical and psychological changes that men generally go through. Every man experiences a decline in bio-available testosterone but some mens levels dip lower than others.

Testosterone begins to decline in men at about age 25. Testosterone levels decline gradually over the years and because it comes on slowly, most men often accommodate to the symptoms and do not realize how much they have lost.

Look at the chart to the right. Where on that testosterone level down turn do you think you are?

Many men, after 35 or so, often have a hard time rising to the occasion and challenge of daily stress. It has only been recently that andropause has received attention and recognition, but why the holdup?

Doctors and scientists are well aware of the ramifications due to the absence of estrogen and progesterone in women. In the mean time, men have kept their focus from themselves and their own hormonal induced weaknesses. Why?

At CORE Medical New York our patients talk openly about their problems and what they are going through. But each of them would also admit that they had difficulty making that first call and that they still cannot admit or talk to their friends about their dysfunctions associated with low testosterone.

Men who receive testosterone therapy consequently report that they feel sexier, stronger and healthier. They say that it makes them feel as they did when they were in their prime.

Testosterone Treatments may stop and reverse the physical decline that robs men of their energy, strength and libido. Testosterone can restore muscle tone and improve stamina. Testosterone can restore healthy sexual excitement and desire, which in turn, results in an improvement in mood and overall well being.

Restoring testosterone to youthful levels with testosterone replacement can reverse the situation. All too often, men automatically assume that as they age, their sexual capacity will diminish. There is no need to accept this loss of sexuality. We should be able to live our life with the same excitement and enthusiasm we enjoyed during our youth.

Potential Testosterone Therapy results:

GET STARTED NOW!

CALL US AT (844) NYC-CORE

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Hormone Replacement Clinic NY, Testosterone Injections for …

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The Hormone Reset Diet – Order the book

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When your metabolism is broken, you get fatter no matter what you doespecially after age forty. The Hormone Reset Diet will show you, in seven three-day bursts, how to make specific dietary changes, eliminate metabolism-wrecking foods (meat and alcohol, sugar, fruit, caffeine, grain, dairy, and toxins), repair your body and reset your hormones.

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The Hormone Reset Diet – Order the book

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Chinese scientists already used Crispr gene editing on 86 …

China is taking the lead in the global race to perfect gene therapies.

Scientists have genetically engineered the cells of at least 86 cancer and HIV patients in the country using Crispr-Cas9 technology since 2015, the Wall Street Journal reports (paywall). Although no formal scientific papers have been written about these experiments, doctors told journalists at the WSJ that some patients have improved. There have also been least 15 deaths, seven of which were in one trial. Scientists report all of these deaths were related to patients previous conditions and not Crispr treatment.

These therapies, which involved taking the immune cells from hospital patients, editing the cells, and transfusing them back into the body, are the first to use Crispr-Cas9 in living humans.

In 2013 scientists first used (paywall) Crispr on on human DNA, and in 2017, US scientists at Oregon Health & Science University reported using the technology to edit human embryos. (The embryos were not allowed to develop further.) It took two years for the Oregon team to receive ethical approval for their experiment. It took the same amount of time for the University of Pennsylvania hospital and the US Food and Drug Administration to give Penn researchers the go-ahead to test a Crispr-based therapy on 18 cancer patients. That trial is expected to begin later this year. Scientists at the Cambridge, Massachusetts-based Crispr Therapeutics also hope to start phase I clinical trials using Crispr to treat patients with a genetic disorder called beta-thalassemias.

Crispr trials on humans have been relatively slow to develop in the US and UK in part due to concerns over how the risk of the procedure is communicated to patients. The Penn scientists first had to consult with an advisory board from the National Institutes of Health set up specifically to evaluate the potential risks and benefits of Crispr therapies, then get approval from the US Food and Drug Administration.

The FDA approved three gene therapies for treatment in 2017, none of which use Crispr. Two of these therapies treat late-stage forms of cancer, and both rely on editing the patients immune cells. The third, which targets a rare form of childhood blindness, works by modifying cells in the eye.

The Chinese ministry of health has to approve all gene-therapy clinical trials in China, but these regulations appear relatively relaxed. According to the WSJ, at Hangzhou Cancer Hospital, for example, a proposal to test a cancer treatment that modifies patients immune cells was approved in a single afternoon. One member of the hospitals approval committee told the WSJ that she did not really understand the science laid out for her in a 100-page document, but was told that the side effects were mild. This was enough for her to give it the go-ahead.

The truth, though, is that there is a dearth of data on the safety of Crispr on humans, and many scientists in the field are concerned that the treatment may cause unintended mutations or may not work at all.

If any of these Crispr treatments are proven successful under scientific scrutiny, theyd be the first of their kind.

Correction: An earlier version of this article stated that about half of the deaths in Crispr trials were related to the gene therapy. It has been corrected to reflect that doctors say all of the deaths in the Crispr trials were related to patients previous conditions.

Read this next: A highly successful attempt at genetic editing of human embryos has opened the door to eradicating inherited diseases

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Allogeneic Cardiac-Derived Stem Cells for Patients …

This project aims to demonstrate both safety and efficacy of a heart-derived cell product in patients who have experienced a heart attack either recently or in the past by conducting a mid-stage (Phase II) clinical trial. The cell product is manufactured using heart tissue obtained from a healthy donor and can be used in most other individuals. Its effect is thought to be long-lasting (months-years) although it is expected to be cleared from the body relatively quickly (weeks-months). Treatment is administered during a single brief procedure, requiring a local anesthetic and insertion of a tube (or catheter) into the heart. The overriding goal for the product is to prevent patients who have had a heart attack from deteriorating over time and developing heart failure, a condition which is defined by the hearts inability to pump blood efficiently and one which affects millions of Americans. At the outset of the project, a Phase I trial was underway. The Phase II trial was initiated at the beginning of the current reporting period, and all subjects enrolled in Phase I completed follow up during the current reporting period. Fourteen patients were treated with the heart-derived cell product as part of Phase I. The safety endpoint for the trial was pre-defined and took into consideration the following: inflammation in the heart accompanied by an immune response, death due to abnormal heart rhythms, sudden death, repeat heart attack, treatment for symptoms of heart failure, need for a heart assist device, and need for a heart transplant. Both an independent Data and Safety Monitoring Board (DSMB) and CIRM agreed that Phase I met its safety endpoint. Preliminary efficacy data from Phase I collected during the current reporting period showed evidence of improvements in scar size, a measure of damage in the heart, and ejection fraction, a measure of the hearts ability to pump blood. At the end of the current reporting period, Phase II is still enrolling subjects and clinical trial sites are still being brought on for participation in the trial. Meanwhile, the manufacturing processes established continue to be employed to create cell products for use in Phase II. Manufacturing data and trial status updates were also provided to the Food and Drug Administration (FDA) as part of standard annual reporting.

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Crisprs Next Big Challenge: Getting Where It Needs to Go | WIRED

Your DNA is your bodys most closely guarded asset. To reach it, any would-be-invaders have to get under your skin, travel through your bloodstream undetected by immune system sentries, somehow cross a cell membrane, and finally find their way into the nucleus. Most of the time, thats a really good thing. These biological barriers prevent nasty viruses from turning your cells into disease-making factories.

But theyre also standing between patients with debilitating genetic diseases and their cures. Crispr, the promising new gene editing technology, promises to eradicate the world of human sufferingbut for all the hype and hope, it hasnt actually cured humans of anything, yet. Medical researchers have the cargo, now they just have to figure out the delivery route.

The first US trials of Crispr safety are set to begin any day now, with Europe expected to follow later this year. Chinese scientists, meanwhile, have been testing Crispr humans since 2015, as The Wall Street Journal recently reported, with mixed success. These first clinical forays involve removing cells from patients bodies, zapping them with electricity to let Crispr sneak in, then infusing them back into their bodies, to either better fight off cancer or to produce a missing blood protein. But that wont work for most rare genetic diseasesthings like cystic fibrosis, Duchennes muscular dystrophy, and Huntingtons. In the 34 trillion-cell sea that is your body, an IV bag full of Crisprd cells simply wont make a dent.

This is the same problem that has plagued the stop-and-go field of gene therapy for nearly three decades. Traditional gene therapy involves ferrying a good copy of a gene inside a harmless virus, and brute-forcing it into a cells DNA. Crisprs cutting action is much more elegant, but its bulk and vulnerability to immune attacks make it just as difficult to deliver.

The challenge is getting gene editors to the right place at the right time in the right amount, says Dan Anderson, an MIT chemical engineer and one of the scientific founders of Crispr Therapeutics. Thats a problem people have been working on for a long time. As of today there certainly is no one way to cure every disease with a single delivery formulation.

And its unlikely there will be anytime soon. So for now, most Crispr companies are taking more of a whatever works approach, borrowing mostly from gene therapys few success stories. One of those is a small, harmless helper virus called AAV, well-suited for carrying genetic instructions into a living cell. AAV wont make you sick, but it can still sneak into your cells and hijack their machinery, making them a perfect Trojan horse in which to put good stufflike a correct copy of a gene, or instructions for how to make the protein-RNA pair that forms the Crispr complex. Crisprs instructions are quite long, so they often cant fit inside one virus.

But once you get around that, theres an even bigger downside to AAV; once it ferries Crispr inside a cell, theres no good way to control its expression. And the longer Crispr hangs around, the greater the chance it could make unwanted cuts.

Delivering Crispr into the cell directly, as opposed to teaching the cell to build it, would provide more control. But doing that means enveloping the unwieldy, charged protein complex in a coating of fat particlesone that can simultaneously shield it from the immune system, get it across a cell membrane, and then release it to do its cutting work unencumbered. Although the technology is improving, its still not very efficient.

The big threeCrispr Therapeutics, Editas Medicine, and Intellia Therapeuticsas well as the latest newcomer, Casebia, are all investing in AAV and lipid nanoparticles, and testing both for their first rounds of treatment. Were leveraging existing delivery technologies, while exploring and developing the next generation, says Editas CEO Katrine Bosley. We will use whatever works best for a given target.

But industry isnt the only one feeling the urgency. This week the National Institutes of Health announced it will be awarding $190 million in research grants over the next six years, in part to push gene editing technologies into the mainstream. The focus of the Somatic Cell Genome Editing program is to dramatically accelerate the translation of these technologies to the clinic for treatment of as many genetic diseases as possible, NIH Director Francis Collins said in a statement Tuesday. Which could encourage some of the more exotic, experimental delivery systems out in the research worldstrategies like Crispr-covered gold beads, yarn-like ball structures called DNA nanoclews, and shape-shifting polymers to get the editor where it needs to go.

In October, UC Berkeley researchers Kunwoo Lee, Hyo Min Park, and Nirhen Murthy used those gold nanoparticles to repair the muscular dystrophy gene in mice. Theyre now expanding that work in a startup the trio cofounded called GenEdit. They plan to develop a suite of nanoparticle delivery vehicles optimized to different tissues, starting with muscles and the brain. Then theyll partner with the folks making the Crispr payloads. That will make it the first company devoted solely to Crispr delivery. The gene editing world is filling up with products to deliverbut even Amazon needs UPS.

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Crisprs Next Big Challenge: Getting Where It Needs to Go | WIRED

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Stem Cell Center Of NJ – New Jersey Stem Cell Therapy

COPD

Over 32 million Americans suffer from chronic obstructive pulmonary disease (also known as COPD). COPD is a progressive lung disease, however regenerative medicine, such as lung regeneration therapies using stem cells are showing potential for COPD by encouraging tissue repair and reducing inflammation to the diseased lung tissue.

Following up with stem cell therapy and exome therapy immediately in the first 36 to 48 hours after stroke symptoms surface has proven to be crucial to long-term recovery and regaining mobility again. Cell therapy also calms post-stroke inflammation in the body, and reduces risk of serious infections.

Parkinsons is a neurodegenerative brain disorder caused by the gradual loss of dopamine-producing cells in the brain. It afflicts more than 1 million people in the U.S., and currently, there is no known cure. Stem cell therapies have been showing incredible progress. Using induced pluripotent stem (iPS) cells, a mature cell can be reprogrammed into an embryonic-like, healthy and highly-functioning state, which has the potential to become a dopamine-producing cell in the brain.

A thick, full head of hair is possible, naturally! Stem cell and exosome therapy promotes healing from within to naturally stimulate hair follicles, which encourages new hair growth. Using your own stem cells, Platelet Rich Plasma (PRP) and exosomes, you can regrow your own healthy, thick hair naturally and restore your confidence!

Erectile Dysfunction (ED) is the inability to achieve or maintain an erection sufficient for satisfactory sexual intercourse. Regenerative medicine offers a non-surgical option that commonly uses the patients own stem cells, exosomes, and other sources of growth factors to regenerate healthy tissue to improve performance and sensation.

If chronic joint pain is derailing your active lifestyle, then youre not alone. Regenerative medicine offers a non-surgical option that commonly uses the patients own stem cells, exosomes, and other sources of growth factors to reduce inflammation, promote natural healing and regenerate healthy tissue surrounding the joint for relief.

Multiple Sclerosis (MS) affects 400,000 people in the U.S., and occurs when the body has an abnormal immune system response and attacks the central nervous system. Regenerative medicine now offers treatment for MS with stem cell therapy, which is an exciting and rapidly developing field of therapy. Stem cells work to repair damaged cells these new cells can become replacement cells to restore normal functionality.

Spinal cord injuries are as complex as they are devastating. Today, cellular treatments, usually a combination of therapies, such as stem cell, Platelet Rich Plasma (PRP) and exosome therapy with growth factors are showing promise in contributing to spinal cord repair and reducing inflammation at the site of injury.

If you have chronic nerve injury pain that doesnt fade, your health care provider may recommend surgery to reverse the damage. However, regenerative medicine offers a non-surgical option to repair damaged tissue and reduce inflammation at the site of injury. Stem cell therapy commonly uses the patients own stem cells, exosomes, and other sources of growth factors to regenerate healthy tissue.

Neuropathy also called peripheral neuropathy occurs when nerves are damaged and cant send messages from the brain and spinal cord to the muscles, skin and other parts of the body. Simply put, the two areas stop communicating. Stem cell and exosome therapies treat damaged nerves affected by neuropathy, and they have the ability to replicate and create new, healthy cells, while repairing damaged tissue.

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Stem Cell Center Of NJ – New Jersey Stem Cell Therapy

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Pluripotent Stem CellBased Therapy for Heart Disease …

Five million people in the U.S. suffer with heart failure, resulting in ~60,000 deaths/year at a cost of $30 billion/year. Heart failure occurs when the heart is damaged and becomes unable to meet the demands placed on it. Unlike other organs, the heart is unable to fully repair itself after injury. One of the common causes for the development of heart damage is a heart attack. After a myocardial infarction (heart attack), irreversible loss of contracting heart muscle cells occurs, resulting in scar formation and subsequently heart failure. Current therapies designed to treat heart attack patients in the acute setting include medical therapies and catheter-based technologies that aim to open the blocked coronary arteries with the hope of salvaging as much of the jeopardized heart muscle cells as possible. Unfortunately, despite advances over the past 2 decades, it is rarely possible to rescue the at-risk heart muscle cells from some degree of irreversible injury and death.

Attention has turned to new methods of treating heart attack and heart failure patients in both the acute and chronic settings after their event. Heart transplantation remains the ultimate approach to treating end-stage heart failure patients but this therapy is invasive, costly, some patients are not candidates for transplantation given their other co-morbidities, and most importantly, there are not enough organs for transplanting the increasing number of patients who need this therapy. As such, newer therapies are needed to treat the millions of patients with debilitating heart conditions. Recently, it has been discovered that stem cells may hold therapeutic potential for these patients. Experimental studies in animals have revealed encouraging results when pluripotent stem cells are introduced into the heart around areas of myocardial infarction. These therapies appear to result in improvement in the contractile function of the heart.

However, numerous questions remain unanswered concerning the use of pluripotent stem cells as therapy for patients with heart attack and heart failure. Human embryonic stem (ES) cells and induced pluripotent stem (iPS) cells grow and divide indefinitely while maintaining the potential to develop into many tissues of the body, including heart muscle. They provide an unprecedented opportunity to both study human heart muscle in culture in the laboratory, and advance the possibility of their use in therapy for damaged heart muscle. We have developed methods for identifying and isolating specific types of human ES and iPS cells, stimulating them to become human heart muscle cells, and delivering these into the hearts of rodents that have had a heart attack. This research will refine and advance such approaches in small and large animals, develop clinical grade cells for use, and ultimately initiate clinical trials for patients suffering from heart disease.

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Pluripotent Stem CellBased Therapy for Heart Disease …

Recommendation and review posted by simmons

Healthier Andropause Build to Balance

To the men out there, this post is for you! Im sure youre well aware that testosterone is critical for your health and vitality. But are you aware of its decline during the aging process, and that you can start doing something about it today with healthy diet and lifestyle practices?

Andropause, sometimes referred to as Male Menopause or Hypogonadism, is the decline of testosterone production in aging men (starting around 50s). Testosterone is a male sex hormone that is important for sexual and reproductive development. The hormone influences sex drive, sperm production, fat distribution, red cell production, maintenance of muscle strength and mass, and the prevention of osteoporosis in men. When its production starts to decline, primarily due to aging, men can experience unfavorable symptoms (many similar to menopause):

Aging is an inevitable part of life, and a top contributing factor for andropause. SHBG (sex hormone binding globulin) increases with age, which binds with testosterone rendering it unavailable. A healthy diet and lifestyle can help slow the aging process and the onset of andropause. Here is what to focus on:

Bottom line keep that male vitality going strong today and every day by adopting a healthy diet and lifestyle.

References:

Bauman, E. NC202.2 Mens & Womens Health Lecture 2 (PowerPoint Handout). Retrieved from Bauman College: https://baumancollege.instructure.com

Rettner, R. (June 2017). What is Testosterone? Live Science. Retrieved from https://www.livescience.com/38963-testosterone.html

The Truth About Alcohol, Fat Loss, and Testosterone. (Oct. 2016). Prostate.net.Retrieved fromhttps://prostate.net/articles/join-30-day-alcohol-fast-t-levels-liver-will-thank

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Healthier Andropause Build to Balance

Recommendation and review posted by sam

Diminished Androgen and Estrogen Receptors and Aromatase …

Aims: One third of males with type 2 diabetes have hypogonadism, characterized by low total and free testosterone concentrations. We hypothesized that this condition is associated with a compensatory increase in the expression of androgen receptors (AR) and that testosterone replacement reverses these changes. We also measured estrogen receptor and aromatase expression.

Materials and Methods: This is a randomized double-blind placebo controlled trial. 32 hypogonadal and 32 eugonadal men with type 2 diabetes were recruited. Hypogonadal men were randomized to receive intramuscular testosterone or saline every 2 weeks for 22 weeks. We measured AR, ER and aromatase expression in peripheral blood mononuclear cells (MNC) and adipose tissue in hypogonadal and eugonadal males with type 2 diabetes at baseline and after 22 weeks of treatment in those with hypogonadism.

Results: The mRNA expression of AR, ER and aromatase in adipose tissue from hypogonadal men was significantly lower as compared to eugonadal men and it increased significantly to levels comparable to those in eugonadal patients with type 2 diabetes following testosterone treatment. AR mRNA expression was also significantly lower in MNC from hypogonadal patients compared to eugonadal T2DM patients. Testosterone administration in hypogonadal patients also restored AR mRNA and nuclear extract protein levels from MNC to that in eugonadal patients.

Conclusions: We conclude that, contrary to our hypothesis, the expression of AR, ER and aromatase is significantly diminished in hypogonadal men as compared to eugonadal men with type 2 diabetes. Following testosterone replacement, there is a reversal of these deficits.

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Diminished Androgen and Estrogen Receptors and Aromatase …

Recommendation and review posted by Bethany Smith

Attention, Men: Doing This Every Day Could Lower Your …

Got a headache? Take an ibuprofen. Sore muscles? Pop a painkiller. But if you take a daily dose of ibuprofen, we have some bad news: All of those pills can add up. They might increase your risk ofheart attacksandcausemuscle weakness, for starters. Now, new research shows that ibuprofen can damage fertility, too.

According to a new study published in Proceedings of the National Academy of Sciences, men who take this popular pain reliever over a long period of time might be more likely to develop a condition called compensated hypogonadism, which could reduce their fertility. Find out the other daily habits that may be harming your fertility.

For the study, 31 men between the ages of 18 and 35 took 600 milligrams (three tablets) a day of ibuprofen for six weeks. Other volunteers received a placebo drug. Then, a team of researchers from Denmark and France monitored the participants for two weeks.

By the end of the study, all of the volunteers showed higher levels of luteinizing hormones, which prevented certain cells in their testicles from producing testosterone. The researchers also found that participants’ pituitary glands were producing more of another hormone that encouraged their bodies to produce more testosterone.

While the combination of these two responses kept the participants’ overall testosterone levels constant, the changes still overworked their bodies, causing compensated hypogonadism. This condition can cause a temporary reduction in the production of sperm cells in men, reducing their fertility.

But hold upyou might not want to toss those painkillers just yet. It’s likely that the average ibuprofen user won’t experience any negative side effects to their fertility; on the other hand, regularly using the drug for long periods of time could be cause for concern, researchers say. Still, it cant hurt to cut back on the pills in the meantime, regardless of your normal doseat least until further studies are done.

Concerned about your baby-making ability? Heres what men can do to boost their fertility.

[Source: MedicalXpress]

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Attention, Men: Doing This Every Day Could Lower Your …

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