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Why are Adult Stem Cells Important? Boston Children’s …

Adult stem cells are the bodys toolbox, called into action by normal wear and tear on the body, and when serious damage or disease attack. Researchers believe that adult stem cells also have the potential, as yet untapped, to be tools in medicine. Scientists and physicians are working towards being able to treat patients with their own stem cells, or with banked donor stem cells that match them genetically.

Grown in large enough numbers in the lab, then transplanted into the patient, these cells could repair an injury or counter a diseaseproviding more insulin-producing cells for people with type 1 diabetes, for example, or cardiac muscle cells to help people recover from a heart attack. This approach is called regenerative medicine.

A number of challenges must be overcome before the full therapeutic potential of adult stem cells can be realized. Scientists are exploring practical ways of harvesting and maintaining most types of adult stem cells. Right now, scientists do not have the ability to grow the cells in the amounts needed for treatment. More work is also needed to find practical ways to direct the different kinds of cells to where theyre needed in the body, preferably without the need for surgery or other invasive methods.

Research in all aspects of adult stem cells and their potential is underway at Childrens Hospital Boston. Realizing that potential will require years of research, but promising strides are being made.

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Creating Embryonic Stem Cells Without Embryo Destruction

By: Ian Murnaghan BSc (hons), MSc - Updated: 12 Sep 2015| *Discuss

One of the biggest hurdles in stem cell research involves the use of embryonic stem cells. While these stem cells have the greatest potential in terms of their ability to differentiate into many different kinds of cells in the human body, they also bring with them enormous ethical controversies. The extraction of embryonic stem cells involves the destruction of an embryo, which upsets and outrages some policy makers and researchers as well as a number of public members. Not only that, but actually obtaining them is a challenge in itself and one that has become more difficult in places such as the United States, where policies have limited the availability of embryonic stem cells for use.

Although researchers have focused on harnessing the power of adult stem cells, there have still been many difficulties in the practical aspects of these potential therapies. In an ideal world, we would be able to use embryonic stem cells without destroying an embyro. Now, however, this ideal hope may actually have some realistic basis. In recent medical news, there has been important progress in the use of embryonic stem cells.

There are still many more tests and research that must be conducted to verify the safety and reliability of the procedure but it is indeed hopeful that funding can now increase for stem cell research. If you are an avid reader of health articles, you will probably be able to stay up-to-date on the latest developments related to this medical news. This newest research into embryonic stem cells holds promise and hope for appeasing the controversy around embryonic stem cell use and allowing for research to finally move forward with fewer challenges and controversies. For those who suffer from one of the many debilitating diseases and conditions that stem cell treatments may help or perhaps cure one day, this is welcome news.

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Disease Prevention & Treatment 5th Edition: Life Extension …

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ISBN-13: 978-0965877787

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Frequently Asked Questions | Cryonics Institute

Good news: you heard wrong! With CI, the minimum fee for cryopreservation at CI (which includes vitrification perfusion and long term storage) is $28,000 a one-time fee, due at time of death. And though the fee can be paid in cash, usually a member has a life insurance policy made that pays the amount to CI upon death. A term life insurance policy in the amount of the minimum fee often costs around $30 per month for a person starting their policy in good health at middle age. Funding at a higher level can be used to defray additional costs, including transportation (which is not included in CIs base fee) or even a cryonics standby team to perform rapid cooling and cardiopulmonary support upon pronouncement of death.

Advice from an insurance professional is recommended before selecting a policy.

A person who wishes to become a Lifetime CI Member can make a single membership payment of $1,250 with no further payment required. If a new member would rather pay a smaller amount up front, in exchange for funding a slightly higher cryopreservation fee later on ($35,000), he or she can join with a $75 initiation fee, and pay annual dues of only $120, which are also payable in quarterly installments of $35. (And such a dues-paying member can upgrade to Lifetime Membership at any time, saving $7,000 and future any dues.) Members at a distance may have to pay local funeral director fees and transportation costs to Michigan to be cryopreserved. These payments are not made to CI, and are not included in the figures outlined above.

Take a look at our Membership FAQ and the membership application forms to find out more. And if you've got any questions, or want to talk about making special arrangements? Give us a call at (586) 791-5961 or drop us an email at We're more than happy to help.

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International Cell and Gene therapy Conferences | Gene …

About Conference

About Conference

In continuation to 1st successful past scientific meeting, 2nd EuroSciCon Conference on Cell and Gene Therapy will be held on April 08-09, 2019 on Paris, France.

EuroSciCon suggests every single person to attend "Genetherapy 2019 in the midst of April 08-09, 2019 at Paris, France which merges brief keynote introductions, speaker talks, Exhibitions, Symposia, Workshops.

Genetherapy 2019 will gather world-class educators, researchers, analysts, Molecular Biologist, Gene therapists , Young Researchers working in the related fields to consider, exchange views and their experiences before an extensive worldwide social occasion of individuals. The social gathering warmly welcomes Presidents, CEO's, Delegates and present day experts from the field of Gene therapy and Public wellbeing and other pertinent organization positions to take an interest in these sessions, B2B get together and board talks. The assembly of this event will be revolving around the topic Exploring the possibilities and breakthroughs in cell and gene therapy.

EuroSciCon is the longest running independent life science events company with a predominantly academic client base. Our multi professional and multi-specialty approach creates a unique experience that cannot be found with a specialist society or commercially. EuroSciCon are corporate members of the following organizations: Royal Society of Biology, IBMS Company.

This global meeting gives the chance to Molecular Biologist, Gene Therapists, young researchers, specialists and analysts throughout the world to assemble and take in the most recent advances in the field of Cell and Gene Therapy and to trade innovative thoughts and encounters.

2 days of scientific exchange

100+ abstracts submitted

20+ scientific sessions

50+ worldwide professionals

80+ healthcare experts

Genetherapy 2019 is the yearly gathering directed with the help of the Organizing Committee Members and individuals from the Editorial Board of the supporting cell and Gene therapy related journals.

Reason to attend?

Genetherapy 2019 is relied upon to give young researchers and scientists a platform to present their revolutions in the field of Cell and Gene Therapy. This conference invites Presidents, CEO's, Delegates and present day specialists from the field of Cell and Gene Therapy and Public wellbeing and other pertinent organization positions to take an interest in this sessions, B2B get together and board talks.

About City:

Paris, the world's most popular city destination, has plenty of must-see places but make sure you spend at least a day strolling off the beaten path, as this is the only way to discover the real Paris: a lively cosmopolitan but undeniably French city.

The city is known for its cafe culture and designer boutiques along the Rue du Faubourg Saint-Honor. Paris is the city of love, inspiration, art and fashion. It has a population of more than 2million people and is divided into 20 districts. Paris has a lot of interesting architecture and museums to offer; among them the famous tourist place to visit is the Eiffel Tower. A significant number of the acclaimed roads and city building areas structures where changed by Haussmann and Napoleon III (Charles Louis Napoleon Bonaparte). The lanes where made much wider, places and squares where fabricated and the structures totally modified. Paris has a nickname called La Ville-Lumiere. The famous places to visit in Paris are Notre Dame Cathedralwhich is Roman Catholic Cathedral situated in the eastern half of the city, Louvre Museum which is located at the heart of Paris , Champs Elysees which is a Arc of Triumph, Montmartre which is a hill located at the north of Paris and its height is 130 metre, it is best known White Domed Basilica of the sacred heart at the top, Quartier Latin which is called the famous private garden located on the left bank of the seine around the Sorbonne, Disneyland Paris which is located 32 km from central Paris , it has two theme parks Disneyland and Walt Disney studios.

Track 1: Cell Science Research

Cell Science Research examines cells their physiological properties, their structure, the organelles they contain, interchanges with their condition, their life cycle, division, end and cell work.

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Track 2: Cell & Gene Therapy

Quality treatment is described as a plan of approaches that modify the announcement of a man's characteristics or repair bizarre characteristics. Each system incorporates the association of a specific nucleic destructive (DNA or RNA). Nucleic acids are frequently not taken up by cells, henceforth exceptional transporters; implied 'vectors' are required. Vectors can be of either mainstream or non-viral nature however Cell treatment is portrayed as the association of living whole cells into the patient for the treatment of a disease. The start of the cells can be from a comparable individual (autologous source) or from another individual (allogeneic source). Cells can be gotten from undifferentiated life forms, for instance, bone marrow or induced pluripotent central microorganisms (iPSCs), rethought from skin fibroblasts or adipocytes. Youthful microorganisms are associated with respect to bone marrow transplantation particularly. Distinctive methods incorporate the utilization of basically create cells, isolated in vitro (in a dish) from essential microorganisms.

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Track 3: Regenerative Medicine

Regenerative Medicine implies a social affair of biomedical approaches to manage investigate and clinical applications which are away to supplant or "recouping" human cells, tissues or organs to restore or set up conventional limits which were vexed on account of afflictions. The field of Regenerative medication has pulled in much thought as it holds the assurance of recuperating hurt tissues and organs in the body by supplanting hurt tissue or by strengthening the body's own repair segments to patch hurt tissues or organs. It in like manner may enable analysts to create tissues and organs in the lab and safely install them inside the body. Regenerative courses of action subsequently can be a dynamic progress in the field of therapeutic administrations.

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Track 4: Immunotherapy

Due to rapidly pushing field of tumor immunology as of late, there has been age of a couple of new procedures for treating development called Immunotherapies. Immunotherapy is a sort of treatment that extends the nature of safe response against tumors either by enabling the activities of specific sections of safe structure or by checking signals conveyed by illness cells that cover safe responses. A couple of sorts of immunotherapy are also called as biologic treatment or biotherapy. Late movements in development immunotherapies have given new supportive systems. These consolidate tumor-related macrophages as treatment centers in oncology, in-situ commencement of platelets with checkpoint inhibitors for post-careful development immunotherapy, safe checkpoint blockade and related endocrinopathies and some more.

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Track 5 : Genetics and stem cell biology

An undifferentiated mass of cell in a multicellular animal which is prepared for offering rise to uncertain number of cells of a comparable sort, and from which certain diverse sorts of cell rise by detachment. Undifferentiated life forms can isolate into specific cell creates. The two describing characteristics of an undifferentiated cell are endless self-restoration and the ability to isolate into a specific adult. There are two critical classes of youthful microorganisms: pluripotent that can end up being any cell in the adult body, and multipotent that is kept to transforming into a more limited masses of cells.

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Track 6: Epigenetics

The examination of changes in living creatures caused by alteration of quality verbalization instead of adjustment of the inherited code itself. Epigenetics are unfaltering heritable characteristics that can't be cleared up by changes in DNA progression.

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Track 7: Human Genomics

The human genome is the total arrangement of nucleic corrosive groupings for people, encoded as DNA inside the 23 chromosome combines in cell cores and in a little DNA particle found inside individual mitochondria. Human genomes incorporate both protein-coding DNA genes and noncoding DNA

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Track 8: Next Generation Sequencing

Deoxyribonucleic destructive, for the most part known as DNA, contains the outlines of life. Inside its structures are the codes required for the party of proteins and non-coding RNA these sub-nuclear mechanical assemblies impact all the natural systems that make and care forever. By understanding the game plan of DNA, examiners have had the ability to outline the structure and limit of proteins and what's more RNA and have gotten a cognizance of the essential purposes behind ailment. Front line Sequencing (NGS) is an able stage that has enabled the sequencing of thousands to countless iotas in the meantime. This compelling device is evolving fields, for instance, redid medicine, inherited infections, and clinical diagnostics by offering a high throughput elective with the capacity to progression various individuals meanwhile.

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Track 9: Gene Editing and CRISPR Based Technologies

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) Technology is a champion among the most fit yet clear mechanical assembly for genome changing. It urges and empowers investigators to easily change DNA groupings and modify quality limits. It has various potential applications that join helping innate disseminates, treating and keeping the spread of diseases and improving yields. CRISPR broadly used as CRISPR-Cas9 where CRISPRs are particular stretches out of DNA and Cas9 is the protein which is an aggravate that exhibitions like a few nuclear scissors, fit for cutting DNA strands. The assurance of CRISPR advancement anyway raises moral stresses as it isn't 100% compelling. Regardless, the progression of CRISPR-Cas9 has disturbed the designed science industry these days, being a clear and great quality changing device.

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Track 10: Proteomics

Proteomics is the broad scale examination of proteomes. A proteome is a course of arrangement of proteins made in a living being, structure, or regular setting. We may imply, for instance, the proteome of a creature composes (for example, Homo sapiens) or an organ (for example, the liver). The proteome isn't relentless; it fluctuates from cell to cell and changes after some time. To some degree, the proteome reflects the key transcriptome. Regardless, protein activity (regularly reviewed by the reaction rate of the systems in which the protein is incorporated) is similarly changed by various components despite the verbalization level of the appropriate quality.

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Track 11: Viral Gene Therapy

Customary strategies for quality treatment fuse transfection. It twisted up clearly inefficient and confined fundamentally in view of movement of value into right now duplicating cells invitro. Quality treatment utilizes the transport of DNA into cells by techniques for vectors, for instance, natural nanoparticles or viral vectors and non-viral systems. The Several sorts of contaminations vectors used as a piece of value treatment are retrovirus, adenovirus, disease adeno-related and herpes simplex contamination. While other recombinant viral vector structures have been delivered, retroviral vectors remain the most surely understood vector system for quality treatment traditions and most prominent application on account of their certain significance as the essential vectors made for compelling quality treatment application and the soonest phases of the field of value treatment.

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Track 12: Cell Therapy of Cardiovascular Disorders

Cardiovascular contaminations have transformed into a growing clinical issue all around. the other test in the treatment of the cardiovascular disease is cell transplantation or cell cardiomyoplasty. Exceptional ischaemic harm and relentless cardiomyopathies incite unending loss of cardiovascular tissue and in the end heart disillusionment. Force medications wide mean to tighten the over the top changes that happen when harm and to cut back shot segments of vas diseases. Regardless, they don't improve the patient's close to home fulfillment or the figure more than coordinate. Unmistakable sorts of undifferentiated living beings have been used for primary microorganism treatment.

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Track 13: Regulatory and Safety Aspects of Cell and Gene Therapy

Cell treatment things require a combination of prosperity examinations. Comparable living being and quality things are heterogeneous substances. There are a few zones that particularly ought to be tended to as it is extremely not the same as that of pharmaceuticals. These range from making group consistency, thing soundness to thing prosperity, quality and sufficiency through pre-clinical, clinical examinations and displaying endorsement. This review plots the present headings/administers in US, EU, India, and the related challenges in making SCBP with highlight on clinical point.

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Track 14: Markets & Future Prospects for Cell & Gene Therapy

The immense number of associations related with cell treatment has extended development incredibly in the midst of the past couple of years. More than 500 associations have been recognized to be locked in with cell treatment and 305 of these are profiled 291 co-tasks. Of these associations, 170 are related with fundamental microorganisms. The Profiles of 72 academic establishments in the US related with cell treatment close by their business facilitated efforts. Allogeneic development with in excess of 350 clinical preliminaries is prepared to order the commercialization of cell medicines in publicize. Advance R&D in cell and quality treatment is depended upon to bloom given the normally based purposes of intrigue.

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Track 15: Gene therapy for Diseases

Gene therapy is the addition of particular genes at some particular locales into a person's cells or tissues to treat an illness, in which the inadequate or non-working quality is then supplanted with the working quality.

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Track 16: Stem Cell therapy

Stem Cell therapyis the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use. Recent studies are going on for the treatment ofSpinal cordinjury as well. Thus,Stem cell therapyhas a great scope in future as well.

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Track 17: Gene Editing

Gene Editing is where the defective gene is being expelled or supplanted from the genome, in order to change the imperfect type of quality to a working structure. Different methods, for example, gene substitution, gene knock out, gene knock down are utilized for this reason. Additionally, site coordinated mutagenesis has been broadly utilized for gene altering purposes.

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International Cell and Gene therapy Conferences | Gene ...

Recommendation and review posted by Bethany Smith

Stem Cell Key Terms | California’s Stem Cell Agency

En Espaol

The term stem cell by itself can be misleading. In fact, there are many different types of stem cells, each with very different potential to treat disease.

Stem CellPluripotentEmbryonic Stem CellAdult Stem CelliPS CellCancer Stem Cell

By definition, all stem cells:

Pluripotent means many "potentials". In other words, these cells have the potential of taking on many fates in the body, including all of the more than 200 different cell types. Embryonic stem cells are pluripotent, as are induced pluripotent stem (iPS) cells that are reprogrammed from adult tissues. When scientists talk about pluripotent stem cells, they mostly mean either embryonic or iPS cells.

Embryonic stem cells come from pluripotent cells, which exist only at the earliest stages of embryonic development. In humans, these cells no longer exist after about five days of development.

When isolated from the embryo and grown in a lab dish, pluripotent cells can continue dividing indefinitely. These cells are known as embryonic stem cells.

James Thomson, a professor in the Department of Cell and Regenerative Biology at the University of Wisconsin, derived the first human embryonic stem cell lines in 1998. He now shares a joint appointment at the University of California, Santa Barbara, a CIRM-funded institution.

Adult stem cells are found in the various tissues and organs of the human body. They are thought to exist in most tissues and organs where they are the source of new cells throughout the life of the organism, replacing cells lost to natural turnover or to damage or disease.

Adult stem cells are committed to becoming a cell from their tissue of origin, and cant form other cell types. They are therefore also called tissue-specific stem cells. They have the broad ability to become many of the cell types present in the organ they reside in. For example:

Unlike embryonic stem cells, researchers have not been able to grow adult stem cells indefinitely in the lab, but this is an area of active research.

Scientists have also found stem cells in the placenta and in the umbilical cord of newborn infants, and they can isolate stem cells from different fetal tissues. Although these cells come from an umbilical cord or a fetus, they more closely resemble adult stem cells than embryonic stem cells because they are tissue-specific. The cord blood cells that some people bank after the birth of a child are a form of adult blood-forming stem cells.

CIRM-grantee IrvWeissman of the Stanford University School of Medicine isolated the first blood-forming adult stem cell from bone marrow in 1988 in mice and later in humans.

Irv Weissman explains the difference between an adult stem cell and an embryonic stem cell (video)

An induced pluripotent stem cell, or iPS cell, is a cell taken from any tissue (usually skin or blood) from a child or adult and is genetically modified to behave like an embryonic stem cell. As the name implies, these cells are pluripotent, which means that they have the ability to form all adult cell types.

Shinya Yamanaka, an investigator with joint appointments at Kyoto University in Japan and the Gladstone Institutes in San Francisco, created the first iPS cells from mouse skin cells in 2006. In 2007, several groups of researchers including Yamanaka and James Thomson from the University of Wisconsin and University of California, Santa Barbara generated iPS cells from human skin cells.

Cancer stem cells are a subpopulation of cancer cells that, like stem cells, can self-renew. However, these cellsrather than growing into tissues and organspropagate the cancer, maturing into the many types of cells that are found in a tumor.

Cancer stem cells are a relatively new concept, but they have generated a lot of interest among cancer researchers because they could lead to more effective cancer therapies that can treat tumors resistant to common cancer treatments.

However, there is still debate on which types of cancer are propelled by cancer stem cells. For those that do, cancer stem cells are thought to be the source of all cells that make up the cancer.

Conventional cancer treatments, such as chemotherapy, may only destroy cells that form the bulk of the tumor, leaving the cancer stem cells intact. Once treatment is complete, cancer stem cells that still reside within the patient can give rise to a recurring tumor. Based on this hypothesis, researchers are trying to find therapies that destroy the cancer stem cells in the hopes that it truly eradicates a patients cancer.

John Dick from the University of Toronto first identified cancer stem cells in 1997. Michael Clarke, then at the University of Michigan, later found the first cancer stem cell in a solid tumor, in this case, breast cancer. Now at Stanford University School of Medicine, Clarke and his group have found cancer stem cells in colon cancer and head and neck cancers.

Find out More:

Catriona Jamieson talks about therapies based on cancer stem cells (4:32)

Stanford Publication: The true seeds of cancer

UCSD Publication: From Bench to Bedside in One Year: Stem Cell Research Leads to Potential New Therapy for Rare Blood Disorder

Updated 2/16

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Stem Cell Key Terms | California's Stem Cell Agency

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Advance Stem Cell Therapy in India | Stem Cell Treatment …

Plan your Stem Cell Therapy in India with Tour2India4Health Consultants

Stem cell therapy in India is performed by highly skilled and qualified doctors and surgeons in India. Our hospitals have state-of-art equipment that increase success rate of stem cell treatment in India. Tour2India4Health is a medical value provider that offers access to the stem cell therapy best hospitals in India for patients from any corner of the world. We offer low cost stem cell therapy at the best hospitals in India.

Stem cells have the ability to differentiate into specific cell types. The two defining characteristics of a stem cell are perpetual self-renewal and the ability to differentiate into a specialized adult cell type.

Serving as a sort of repair system, they can theoretically divide without limit to replenish other cells for as long as the person or animal is still alive. When a stem cell divides, each "daughter" cell has the potential to either remain a stem cell or become another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell.

There are three classes of stem cells i.e totipotent, pluripotent and multipotent (also known as unipotent).

Many different terms are used to describe various types of stem cells, often based on where in the body or what stage in development they come from. You may have heard the following terms:

Adult Stem Cells or Tissue-specific Stem Cells: Adult stem cells are tissue-specific, meaning they are found in a given tissue in our bodies and generate the mature cell types within that particular tissue or organ. It is not clear whether all organs, such as the heart, contain stem cells. The term adult stem cells is often used very broadly and may include fetal and cord blood stem cells.

Fetal Stem Cells: As their name suggests, fetal stem cells are taken from the fetus. The developing baby is referred to as a fetus from approximately 10 weeks of gestation. Most tissues in a fetus contain stem cells that drive the rapid growth and development of the organs. Like adult stem cells, fetal stem cells are generally tissue-specific, and generate the mature cell types within the particular tissue or organ in which they are found.

Cord Blood Stem Cells: At birth the blood in the umbilical cord is rich in blood-forming stem cells. The applications of cord blood are similar to those of adult bone marrow and are currently used to treat diseases and conditions of the blood or to restore the blood system after treatment for specific cancers. Like the stem cells in adult bone marrow, cord blood stem cells are tissue-specific.

Embryonic Stem Cells: Embryonic stem cells are derived from very early embryos and can in theory give rise to all cell types in the body. While these cells are already helping us better understand diseases and hold enormous promise for future therapies, there are currently no treatments using embryonic stem cells accepted by the medical community.

Induced Pluripotent Stem Cells (IPS cells): In 2006, scientists discovered how to reprogram cells with a specialized function (for example, skin cells) in the laboratory, so that they behave like an embryonic stem cell. These cells, called induced pluripotent cells or IPS cells, are created by inducing the specialized cells to express genes that are normally made in embryonic stem cells and that control how the cell functions.

Embryonic stem cells are derived from the inner cell mass of a blastocyst: the fertilized egg, called the zygote, divides and forms two cells; each of these cells divides again, and so on. Soon there is a hollow ball of about 150 cells called the blastocyst that contains two types of cells, the trophoblast and the inner cell mass. Embryonic stem cells are obtained from the inner cell mass.

Stem cells can also be found in small numbers in various tissues in the fetal and adult body. For example, blood stem cells are found in the bone marrow that give rise to all specialized blood cell types. Such tissue-specific stem cells have not yet been identified in all vital organs, and in some tissues like the brain, although stem cells exist, they are not very active, and thus do not readily respond to cell injury or damage.

Stem cells can also be obtained from other sources, for example, the umbilical cord of a newborn baby is a source of blood stem cells. Recently, scientists have also discovered the existence of cells in baby teeth and in amniotic fluid that may also have the potential to form multiple cell types. Research on these cells is at a very early stage.

Stem cell therapy is the use of stem cells to treat certain diseases. Stem cells are obtained from the patients own blood bone marrow, fat and umbilical cord tissue or blood. They are progenitor cells that lead to creation of new cells and are thus called as generative cells as well.

The biological task of stem cells is to repair and regenerate damaged cells. Stem cell therapy exploits this function by administering these cells systematically and in high concentrations directly into the damaged tissue, where they advance its self-healing. The process that lies behind this mechanism is largely unknown, but it is assumed that stem cells discharge certain substances which activate the diseased tissue. It is also conceivable that single damaged somatic cells, e.g. single neurocytes in the spinal cord or endothelium cells in vessels, are replaced by stem cells. Most scientists agree that stem cell research has great life-saving potential and could revolutionize the study and treatment of diseases and injuries.

Stem cell therapy is useful in certain degenerative diseases like

If stem cell therapy is an option, a detailed treatment plan is prepared depending on the type of treatment necessary. Once the patient has consented to the treatment plan, an appointment is scheduled for bone marrow extraction. Please note that this is a minimally invasive surgical procedure, so it is important that patients do not take any blood-thinning medication in the ten days prior to the appointment. It is necessary for each patient to consult their own doctor before discontinuing this type of medication.

The treatment procedure include:

Bone Marrow Extraction: Bone marrow is extracted from the hip bone by the physicians. This procedure normally takes around 30 minutes. First, local anesthetic is administered to the area of skin where the puncture will be made. Then, a thin needle is used to extract around 150-200 ml of bone marrow. The injection of local anesthetic can be slightly painful, but the patient usually does not feel the extraction of bone marrow.

Isolation, Analysis and Concentration of the Stem Cells in the Laboratory: The quality and quantity of the stem cells contained in the collected bone marrow are tested at the laboratory. First, the stem cells are isolated. Then a chromatographical procedure is used to separate them from the red and white blood corpuscles and plasma. The sample is tested under sterile conditions so that the stem cells, which will be administered to the patient, are not contaminated with viruses, bacteria or fungi. Each sample is also tested for the presence of viral markers such as HIV, hepatitis B and C and cytomegalia. The cleaned stem cells are counted and viability checks are made. If there are enough viable stem cells, i.e. more than two million CD34+ cells with over 80 percent viability, the stem cell concentrate is approved for patient administration.

Stem Cell Implantation: The method of stem cell implantation depends on the patient's condition. There are four different ways of administering stem cells:

Intravenous administration:

It is important to understand that while stem cell therapy can help alleviate symptoms in many patients and slow or even reverse degenerative processes, it does not work in all cases. Based on additional information, patient's current health situation and/or unforeseen health risks, the medical staff can always, in the interest of the individual patient, propose another kind of stem cell transplantation or in exceptional situations cancel the treatment.

Allogeneic Stem Cell Transplantation: Allogeneic stem cell transplantation involves transferring the stem cells from a healthy person (the donor) to your body after high-intensity chemotherapy or radiation. It is helpful in treating patients with high risk of relapse or who didnt respond to the prior treatment. Allogeneic stem cell transplant cost in India is comparatively less when contrasted with alternate nations.

Autologous Stem Cell Transplant: Patients own blood-forming stem cells are collected and then it is treated with high doses of chemotherapy. The high-dose treatment kills the cancer cells. They are used to replace stem cells that have been damaged by high doses of chemotherapy, used to treat the patient's underlying disease.

The side effects of stem cell therapy differ from person to person. Listed below are the side effects of stem cell therapy :

According to the Indian Council of Medical Research, all is considered to be experimental, with the exception of bone marrow transplants. However, the guidelines that were put into place in 2007 are largely non-enforceable. Regardless, stem cell therapy is legalized in India. Umbilical cord and adult stem cell treatment are considered permissible. Embryonic stem cell therapy and research is restricted.

There is about a 60% to 80% overall success rate in the use of stem cell therapy in both India and around the world. However, success rates vary depending on the disease being treated, the institute conducting the procedures, and the condition of the patient. In order to receive complete information you will have to contact the medical institutes and ask specific questions concerning the patient's condition.

Mrs. Selina Naidoo with her Son from Malaysia

Tour2India4Health has proved to be a blessing in disguise for me. A medical tourism company with everything at par with our expectations has given me the most satisfactory and relieving experience of my life. I went to them for my sons surgery who was suffering from a serious illness and stem cell therapy was the only choice I had. Trust it was heart wrenching to leave my son under any hands on the operation table. Nevertheless, courageously I had to because thats what I was here for and thats what could get my son a new and healthy life. Sitting at a corner outside the operation theatre was taking my heartbeats away with every second. Finally, the surgery was over and I was there in front of the doctor with closed eyes. He declared that the surgery was successful and my son is fine but needs some extra care and some cautious post operative measures for recovery. All through our stay in the hospital, everything went on brilliantly and after my son recovered completely, I came back to my home country. Even after that for many months, I received regular calls to verify and virtually monitor the health of my child. Now, its been 5 years and when I see my child today it feels as if no surgery was ever done on him. Thanks to the doctor who treated him and to the entire team of nurses and travel professionals who displayed extra warmth and care. Thanks is just a small word to say as a mother of a child.

India is the most preferable destination for patients who are looking for low cost stem cell therapy. Indian doctors and healthcare professionals are renowned world over for their skills with many of them holding high positions in leading hospitals in US, UK and other countries around the world. There are significant numbers of highly skilled experts in India, including many who have relocated to India after having worked in the top hospitals across the world.

The Cost of stem cell treatment in India are generally about a tenth of the costs in US and are significantly cheaper compared with even other medical travel destinations like Thailand

*The price for the Stem Cell Therapy is an average collected from the 15 best corporate hospitals and 10 Top Stem Cell Experts of India.

*The final prices offered to the patients is based on their medical reports and is dependent on the current medical condition of the patient, type of room, type of therapy, hospital brand and the surgeon's expertise.

We have worked out special packages of the Stem Cell Therapy for our Indian and International patients. You can send us your medical reports to avail the benefits of these special packages.


There are many reasons for India becoming a popular medical tourism spot is the low cost stem cell treatment in the area. When in contrast to the first world countries like, US and UK, medical care in India costs as much as 60-90% lesser, that makes it a great option for the citizens of those countries to opt for stem cell treatment in India because of availability of quality healthcare in India, affordable prices strategic connectivity, food, zero language barrier and many other reasons.

The maximum number of patients for stem cell therapy comes from Nigeria, Kenya, Ethiopia, USA, UK, Australia, Saudi Arabia, UAE, Uzbekistan, Bangladesh.

Cities where top and world renowned Stem Cell Therapy hospitals and clinics situated are :

We have PAN-India level tie ups with TOP Hospitals for Stem Cell Therapy across 15+ major cities in India. We can provide you with multiple top hospitals & best surgeons recommendations for Stem Cell Therapy in India.

India has now been recognized as one of the leaders in medical field of research and treatment. Tour2India4Health Group was established with an aim of providing best medical services to its patients and since then has been working hard in maintaining itself as one of the most professional healthcare tourism providers in India. With a number of world-renowned medical facilities affiliated, we have the resources to offer you the finest medical treatment in India, and help your speedy recovery. Tour2India4Health Group has always believed and practiced providing its patients best surgery and treatment procedure giving a second chance to live a more better and normal life. Our team serves the clientele most comfortable and convenient measures of healthcare services thus, making your medical tour to India very fruitful experience.

Our facilitation:

We has been operating patients from all major countries like USA, United Kingdom, Italy, Australia, Canada, Spain, New Zealand, and Kuwait etc. We have network of selected medical centers, surgeons and physicians around various cities in India, who qualify our assessment criteria to ensure that our core values of Safety, Excellence and Trust are maintained in all our services.

Below are the downloadable links that will help you to plan your medical trip to India in a more organized and better way. Attached word and pdf files gives information that will help you to know India more and make your trip to India easy and memorable one.

Best Stem Cell Therapy in India, Cost of Stem Cell Therapy in India, Stem Cell Therapy Best Hospitals in India, Success Rate of Stem Cell Treatment in India, Stem Cell Therapy Treatment Cost in India, Allogeneic Stem cell Transplant Cost in India, autologous Stem Cell Transplant Cost in India, Stem Cell Therapy in India, Low Cost Stem Cell Therapy India, Stem Cell Benefits in India, Top Stem Cell Centers in India, Best Doctors for Stem Cell Therapy in India, List of Best Stem Cell Treatment Clinics in India, Allogeneic stem cell transplantation, Allogeneic Stem Cell Transplant Cost in India, Autologous Stem Cell Transplant, Autologous Stem Cell Transplant Cost in India

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NIH launches largest-ever study of breast cancer genetics …

News Release

Wednesday, July 6, 2016

Findings could inform breast cancer disparities.

The largest study ever to investigate how genetic and biological factors contribute to breast cancer risk among black women launched today. This collaborative research project will identify genetic factors that may underlie breast cancer disparities. The effort is funded by the National Cancer Institute (NCI), part of the National Institutes of Health.

This effort is about making sure that all Americans no matter their background reap the same benefits from the promising advances of precision medicine.

Douglas R. Lowy, M.D., Acting Director, NCI

The Breast Cancer Genetic Study in African-Ancestry Populations initiative does not involve new patient enrollment but builds on years of research cooperation among investigators who are part of the African-American Breast Cancer Consortium, the African-American Breast Cancer Epidemiology and Risk (AMBER) Consortium, and the NCI Cohort Consortium. These investigators, who come from many different institutions, will share biospecimens, data, and resources from 18 previous studies, resulting in a study population of 20,000 black women with breast cancer.

This effort is about making sure that all Americans no matter their background reap the same benefits from the promising advances of precision medicine. The exciting new approaches to cancer prevention, diagnosis, and treatment ring hollow unless we can effectively narrow the gap of cancer disparities, and this new research initiative will help us do that, said Douglas R. Lowy, M.D., acting director of NCI. Im hopeful about where this new research can take us, not only in addressing the unique breast cancer profiles of African-American women, but also in learning more about the origin of cancer disparities.

Survival rates for women with breast cancer have been steadily improving over the past several decades. However, these improvements have not been shared equally; black women are more likely to die of their disease. Perhaps of most concern is that black women are more likely than white women to be diagnosed with aggressive subtypes of breast cancer. The rate of triple-negative breast cancer, an aggressive subtype, is twice as high in black women as compared to white women.

The exact reasons for these persistent disparities are unclear, although studies suggest that they are the result of a complex interplay of genetic, environmental, and societal factors, including access to health care. Large studies are needed to comprehensively examine these factors, and NCI is supporting several such efforts.

As part of the study, the genomes of 20,000 black women with breast cancer will be compared with those of 20,000 black women who do not have breast cancer. The genomes will also be compared to those of white women who have breast cancer. The project will investigate inherited genetic variations that are associated with breast cancer risk in black women compared to white women. In addition, researchers will examine gene expression in breast cancer tumor samples to investigate the genetic pathways that are involved in tumor development.

This $12 million grant in combination with previous investments should help advance our understanding of the social and biological causes that lead to disparities in cancer among underserved populations, said Robert Croyle, Ph.D., director of NCIs Division of Cancer Control and Population Sciences (DCCPS), which is administering the grant. A better understanding of the genetic contributions to differences in breast cancer diagnoses and outcomes among African-Americans may lead to better treatments and better approaches to cancer prevention.

A number of studies have suggested that genetic factors may influence breast cancer disparities, so were hopeful that this project can help to shed further light on this matter. said Damali Martin, Ph.D., program director for the DCCPS Genomic Epidemiology Branch. Dr. Martins office is working directly with the grant recipients as well as the consortia groups that have been researching black women and breast cancer.

The grant has been awarded to Wei Zheng, M.D., Ph.D., of Vanderbilt University, Nashville, Tennesee; Christopher Haiman, Sc.D., of the University of Southern California, Los Angeles; and Julie Palmer, Sc.D., of Boston University. Additionally, minority scientists from various institutions, including from one Historically Black College and University medical school, are playing an important role in this study, and they have been involved in previous research that this study builds upon. For example, the Southern Community Cohort Study, a contributing study for this grant, represents a 15-year partnership between Vanderbilt and historically black Meharry Medical College in Nashville, Tennessee. In addition, this grant will provide training opportunities for scientists from minority populations.

Support for ongoing research in this area represents NCIs continued commitment to fund a comprehensive portfolio of research aimed at reducing cancer risk, incidence, and mortality, as well as improving quality of life for cancer survivors across all demographic groups.

The National Cancer Institute leads the National Cancer Program and the NIHs efforts to dramatically reduce the prevalence of cancer and improve the lives of cancer patients and their families, through research into prevention and cancer biology, the development of new interventions, and the training and mentoring of new researchers. For more information about cancer, please visit the NCI website at or call NCI's Cancer Information Service at 1-800-4-CANCER.

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

NIHTurning Discovery Into Health

ReferenceBreast Cancer Genetic Study in African-Ancestry Populations, Grant Number R01CA202981


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Serious question about female genetics? | Yahoo Answers

Don't think that either is superior however, each is superior at specific things, obviously.

But here is some fodder for thought. Interesting Genetic Fact:

The female population currently outweighs the male population by 1% or so making the ratio 51% to 49% roughly. However, the male population is predicted to catch up and perhaps surpass the female population only slightly with modern medicine. Why? Because the sex chromosomes, XX for woman and XY for men, carry different genes. The X chromosomes carry large amounts of DNA information while the Y chromosome which is shorter than the X chromosome, only carries a few bits of genetic information such as the gene for becoming male. Essentially, we all start out female! It is the presence of the testis gene, called SRY, that determines the male gender.

Because the X chromosome carries large amounts of genetic information while the Y does not, males are more likely to suffer from disease and abnormalities than women. In genetics, two genes come together to determine a trait. One or both can be dominant or recessive. Disease genes are recessive as are abnormalities but if a male receives a recessive gene for a disease or abnormality, he is likely to express that gene given the lack of extra DNA information from the Y chromosome. Therefore, more male die in infancy than females. Does this make females genetically superior? Modern medicine will help combat early deaths from a genetic standpoint, helping even out the population. Let's not forget that females also develop faster overall, emotionally, physically, and intellectually.

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Gene therapy might be a cure for "bubble boy disease …

They were born without a working germ-fighting system, every infection a threat to their lives. Now eight babies with "bubble boy disease" have had it fixed by a gene therapy made from one of the immune system's worst enemies HIV, the virus that causes AIDS.

Astudyout Wednesday details how scientists turned this enemy virus into a savior, altering it so it couldn't cause disease and then using it to deliver a gene the boys lacked.

"This therapy has cured the patients," although it will take more time to see if it's a permanent fix, said Dr. Ewelina Mamcarz, one of the study leaders at St. Jude Children's Research Hospital in Memphis.

Omarion Jordan, who turns 1 later this month, had the therapy in December to treat severe combined immunodeficiency syndrome, or SCID.

"For a long time we didn't know what was wrong with him. He just kept getting these infections," said his mother, Kristin Simpson. Learning that he had SCID "was just heartbreaking ... I didn't know what was going to happen to him."

Omarion now has a normal immune system. "He's like a normal, healthy baby," Simpson said. "I think it's amazing."

Study results were published by the New England Journal of Medicine. The treatment was pioneered by a St. Jude doctor who recently died, Brian Sorrentino.

SCID is caused by a genetic flaw that keeps the bone marrow from making effective versions of blood cells that comprise the immune system. It affects 1 in 200,000 newborns, almost exclusively males. Without treatment, it often kills in the first year or two of life.

"A simple infection like the common cold could be fatal," Mamcarz said.

The nickname "bubble boy disease" comes from a famous case in the 1970s a Texas boy who lived for 12 years in a protective plastic bubble to isolate him from germs. A bone marrow transplant from a genetically matched sibling can cure SCID, but most people lack a suitable donor. Transplants also are medically risky the Texas boy died after one.

Doctors think gene therapy could be a solution. It involves removing some of a patient's blood cells, using the modified HIV to insert the missing gene, and returning the cells through an IV. Before getting their cells back, patients are given a drug to destroy some of their marrow so the modified cells have more room to grow.

When doctors first tried it 20 years ago, the treatment had unintended effects on other genes, and some patients later developed leukemia. The new therapy has safeguards to lower that risk.

A small study of older children suggested it was safe. The new study tried it in infants, and doctors are reporting on the first eight who were treated at St. Jude and at UCSF Benioff Children's Hospital San Francisco.

Within a few months, normal levels of healthy immune system cells developed in seven boys. The eighth needed a second dose of gene therapy but now is well, too. Six to 24 months after treatment, all eight are making all the cell types needed to fight infections, and some have successfully received vaccines to further boost their immunity to disease.

No serious or lasting side effects occurred.

Omarion is the 10th boy treated in the study, which is ongoing. It's sponsored by the American Lebanese Syrian Associated Charities, the California Institute of Regenerative Medicine, the Assisi Foundation of Memphis and the federal government.

"So far it really looks good," but patients will have to be studied to see if the results last, said Dr. Anthony Fauci, head of the National Institute of Allergy and Infectious Diseases, which helped develop the treatment. "To me, this looks promising."

Rights to it have been licensed by St. Jude to Mustang Bio. Doctors say they have no estimate on what it might cost if it does become an approved treatment.

A similar technique harnessing a modified version of HIV is also being studied as a possible cure for sickle cell anemia, CBS News chief medical correspondent Dr. Jon LaPook reports. In a clinical trial at the National Institutes of Health, nine adults with sickle cell anemia have undergone the gene therapy. So far, all are responding well.

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New Jersey Anti Aging Programs for Women and Men

Healthy Aging Medical Centers of Essex County New Jersey offers a host of advanced treatment programs to help their patients look and feel their very best including Bioidentical Hormone Replacement Therapy, Testosterone Replacement Therapy, Functional Medicine Services, Cosmetic and Medical Aesthetic procedures, and more.

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If you are sick and tired of being sick and tired, dont wait any longer to learn about the benefits of Bioidentical Hormone Replacement Therapy as part of a total wellness program offered by Dr. Rand at Healthy Aging Medical Centers in Essex County New Jersey. Call today and learn more about the amazing possibilities of Anti-Aging medicine from someone who has experienced the benefits first hand, Doctor Johanan Rand, M.D.

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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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CRISPR gene editing - Wikipedia

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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

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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:

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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 using identifier NCT01512888. More information about the companion trial evaluating the treatment in older children and young adults is available using 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

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 Gene Editing Is Coming for the Womb | WIRED

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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


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


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

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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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The report deeply displays the global Hemophilia Gene Therapy Market.

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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) United States Market States and Outlook (2013-2023) Canada Market States and Outlook (2013-2023) Mexico Market States and Outlook (2013-2023) 1.4.2 Europe (Germany, France, UK, Russia and Italy) Germany Market States and Outlook (2013-2023) France Market States and Outlook (2013-2023) UK Market States and Outlook (2013-2023) Russia Market States and Outlook (2013-2023) Italy Market States and Outlook (2013-2023) 1.4.3 Asia-Pacific (China, Japan, Korea, India and Southeast Asia) China Market States and Outlook (2013-2023) Japan Market States and Outlook (2013-2023) Korea Market States and Outlook (2013-2023) India Market States and Outlook (2013-2023) Southeast Asia Market States and Outlook (2013-2023) 1.4.4 South America, Middle East and Africa Brazil Market States and Outlook (2013-2023) Egypt Market States and Outlook (2013-2023) Saudi Arabia Market States and Outlook (2013-2023) South Africa Market States and Outlook (2013-2023) 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


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