GENE THERAPY PROF WAGIH P1
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GENE THERAPY PROF WAGIH P1
By: Asmaa Alhazmi
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Understanding Advances in Gene Therapy
Could a blood cancer patient #39;s own immune cells soon be used to fight their cancer? In chimeric antigen receptor (CAR) gene therapy, T-cells are taken from a...
By: patientpower
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For science and cancer treatment, my gene therapy question.
So I #39;ve had plenty of time to sit and think about it, but I #39;m still me, I just have the genes changed. But a small degree, I suppose like a grain of sand in ...
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For science and cancer treatment, my gene therapy question. - Video
Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene to replace a mutated gene. Other forms involve directly correcting a mutation, or using DNA that encodes a therapeutic protein drug (rather than a natural human gene) to provide treatment. In gene therapy, DNA that encodes a therapeutic protein is packaged within a "vector", which is used to get the DNA inside cells within the body. Once inside, the DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patient's disease.
Gene therapy was first conceptualized in 1972, with the authors urging caution before commencing gene therapy studies in humans. The first FDA-approved gene therapy experiment in the United States occurred in 1990, when Ashanti DeSilva was treated for ADA-SCID.[1] Since then, over 1,700 clinical trials have been conducted using a number of techniques for gene therapy.[2]
Although early clinical failures led many to dismiss gene therapy as over-hyped, clinical successes since 2006 have bolstered new optimism in the promise of gene therapy. These include successful treatment of patients with the retinal disease Leber's congenital amaurosis,[3][4][5][6]X-linked SCID,[7] ADA-SCID,[8][9]adrenoleukodystrophy,[10]chronic lymphocytic leukemia (CLL),[11]acute lymphocytic leukemia (ALL),[12]multiple myeloma,[13]haemophilia[9] and Parkinson's disease.[14] These recent clinical successes have led to a renewed interest in gene therapy, with several articles in scientific and popular publications calling for continued investment in the field.[15][16]
In 2012, Glybera became the first gene therapy treatment to be approved for clinical use in either Europe or the United States after its endorsement by the European Commission.[17][18]
Scientists have taken the logical step of trying to introduce genes directly into human cells, focusing on diseases caused by single-gene defects, such as cystic fibrosis, haemophilia, muscular dystrophy, thalassemia, and sickle cell anemia. However, this has proven more difficult than genetically modifying bacteria, primarily because of the problems involved in carrying large sections of DNA and delivering them to the correct site on the gene. Today, most gene therapy studies are aimed at cancer and hereditary diseases linked to a genetic defect. Antisense therapy is not strictly a form of gene therapy, but is a related, genetically mediated therapy.
The most common form of genetic engineering involves the insertion of a functional gene at an unspecified location in the host genome. This is accomplished by isolating and copying the gene of interest, generating a construct containing all the genetic elements for correct expression, and then inserting this construct into a random location in the host organism. Other forms of genetic engineering include gene targeting and knocking out specific genes via engineered nucleases such as zinc finger nucleases, engineered I-CreI homing endonucleases, or nucleases generated from TAL effectors. An example of gene-knockout mediated gene therapy is the knockout of the human CCR5 gene in T-cells to control HIV infection.[19] This approach is currently being used in several human clinical trials.[20]
Gene therapy may be classified into the two following types:
In somatic gene therapy, the therapeutic genes are transferred into the somatic cells (non sex-cells), or body, of a patient. Any modifications and effects will be restricted to the individual patient only, and will not be inherited by the patient's offspring or later generations. Somatic gene therapy represents the mainstream line of current basic and clinical research, where the therapeutic DNA transgene (either integrated in the genome or as an external episome or plasmid) is used to treat a disease in an individual.
In germ line gene therapy, germ cells (sperm or eggs) are modified by the introduction of functional genes, which are integrated into their genomes. Germ cells will combine to form a zygote which will divide to produce all the other cells in an organism and therefore if a germ cell is genetically modified then all the cells in the organism will contain the modified gene. This would allow the therapy to be heritable and passed on to later generations. Although this should, in theory, be highly effective in counteracting genetic disorders and hereditary diseases, some jurisdictions, including Australia, Canada, Germany, Israel, Switzerland, and the Netherlands[21] prohibit this for application in human beings, at least for the present, for technical and ethical reasons, including insufficient knowledge about possible risks to future generations[21] and higher risk than somatic gene therapy (e.g. using non-integrative vectors).[22] The USA has no federal legislation specifically addressing human germ-line or somatic genetic modification (beyond the usual FDA testing regulations for therapies in general).[21]
Gene therapy utilizes the delivery of DNA into cells, which can be accomplished by a number of methods. The two major classes of methods are those that use recombinant viruses (sometimes called biological nanoparticles or viral vectors) and those that use naked DNA or DNA complexes (non-viral methods).
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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:
Replacing a mutated gene that causes disease with a healthy copy of the gene.
Inactivating, or knocking out, a mutated gene that is functioning improperly.
Introducing a new gene into the body to help fight a disease.
Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently only being tested for the treatment of diseases that have no other cures.
MedlinePlus from the National Library of Medicine offers a list of links to information about genes and gene therapy.
Educational resources related to gene therapy are available from GeneEd.
The Genetic Science Learning Center at the University of Utah provides an interactive introduction to gene therapy.
The Centre for Genetics Education provides an introduction to gene therapy, including a discussion of ethical and safety considerations.
Additional information about gene therapy is available from the National Genetics and Genomics Education Centre of the National Health Service (UK)
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At the forefront of medicine, Gene Therapy brings you the latest research into genetic and cell-based technologies to treat disease. It also publishes Progress & Prospects reviews and News and Commentary articles, which highlight the cutting edge of the field.
Volume 20, No 12 December 2013 ISSN: 0969-7128 EISSN: 1476-5462
2012 Impact Factor 4.321* 70/290 Biochemistry & Molecular Biology 22/159 Biotechnology & Applied Microbiology 33/161 Genetics & Heredity 25/121 Medicine, Research & Experimental
Editors: J Glorioso, USA N Lemoine, UK
*2012 Journal Citation Reports Science Edition (Thomson Reuters, 2013)
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Open
Gene Therapy now offers authors the option to publish their articles with immediate open access upon publication. Open access articles will also be deposited on PubMed Central at the time of publication and will be freely available immediately. Find out more from our FAQs page.
Reviews by top researchers in the field. See the recent Progress and Prospects articles.
Essential topics explored in depth in Gene Therapy Special Issues.
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Better Quality of Life
In His Own Words: Another Patient Lives Because of Gene Therapy
Watch an Amazing Short Film of a Young Girl's Recovery
Our donors have allowed top scientific minds to explore this new and promising avenue of cancer treatment, and their philanthropy is directly linked to the lives saved so far, Barbara Netter, ACGT president, said. Theres a lot more hope than there ever was. Its a very exciting time the beginning of the Golden Age, Netter described real progress being made through the results of research that have patients who faced dire diagnoses instead being in complete remissionnot short-term but for years and counting.
Read the full article at Connecticut Magazine
ACGT Scientific Advisory Council Chair Dr. Savio Woo introduced each of three ACGT Research Fellows who described how they are Achieving Cancer Remission with Cell and Gene Therapies. Dr. Carl June, University of Pennsylvania, Dr. Laurence Cooper, MD Anderson Cancer Center, and Dr. Michel Sadelain of Memorial Sloan-Kettering Cancer Center thanked ACGT for providing the initial funding that has enabled them to bring new therapies to patients. Read more...
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ACGT® - Alliance for Cancer Gene Therapy - National Grants ...
New York, NY (PRWEB) November 13, 2013
Researchers at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai have successfully tested a powerful gene therapy, delivered directly into the heart, to reverse heart failure in large animal models.
The new research study findings, published in November 13 issue of Science Translational Medicine, is the final study phase before human clinical trials can begin testing SUMO-1 gene therapy. SUMO-1 is a gene that is missing in action in heart failure patients.
SUMO-1 gene therapy may be one of the first treatments that can actually shrink enlarged hearts and significantly improve a damaged hearts life-sustaining function, says the studys senior investigator Roger J. Hajjar, MD, Director of the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai and the Arthur & Janet C. Ross Professor of Medicine at Mount Sinai. We are very eager to test this gene therapy in our patients suffering from severe heart failure.
Heart failure remains a leading cause of hospitalization in the elderly. It accounts for about 300,000 deaths each year in the United States. Heart failure occurs when a persons heart is too weak to properly pump and circulate blood throughout their body.
Dr. Hajjar is already on a path toward approval from the Food and Drug Administration to test the novel SUMO-1 gene therapy in heart failure patients. When it begins, the clinical trial will be the second gene therapy treatment designed to reverse heart failure launched by Dr. Hajjar and his Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai.
The first trial, named CUPID, is in its final phases of testing SERCA2 gene therapy. Phase 1 and phase 2a trial results were positive, demonstrating substantial improvement in clinical events.
In that trial, a gene known as SERCA2 is delivered via an inert virus a modified virus without infectious particles. SERCA2 is a gene that produces an enzyme critical to the proper pumping of calcium out of cells. In heart failure, SERCA2 is dysfunctional, forcing the heart to work harder and in the process, to grow larger.
The virus carrying SERCA2 is delivered through the coronary arteries into the heart during a cardiac catheterization procedure. Studies show only a one-time gene therapy dose is needed to restore healthy SERCA2a gene production of its beneficial enzyme.
But previous research by Mount Sinai discovered SERCA2 is not the only enzyme that is missing in action in heart failure. A study published in Nature in 2011 by Dr. Hajjar and his research group showed that the SUMO-1 gene is also decreased in failing human hearts. But SUMO-1 regulates SERCA2as activity, suggesting that it can enhance the function of SERCA2a without altering its levels. A follow-up study in a mouse model of heart failure demonstrated that SUMO-1 gene therapy substantially improved cardiac function.
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Mount Sinai's Novel Gene Therapy Works to Reverse Heart Failure
Nov. 13, 2013 Researchers at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai have successfully tested a powerful gene therapy, delivered directly into the heart, to reverse heart failure in large animal models.
The new research study findings, published in November 13 issue of Science Translational Medicine, is the final study phase before human clinical trials can begin testing SUMO-1 gene therapy. SUMO-1 is a gene that is "missing in action" in heart failure patients.
"SUMO-1 gene therapy may be one of the first treatments that can actually shrink enlarged hearts and significantly improve a damaged heart's life-sustaining function," says the study's senior investigator Roger J. Hajjar, MD, Director of the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai and the Arthur & Janet C. Ross Professor of Medicine at Mount Sinai. "We are very eager to test this gene therapy in our patients suffering from severe heart failure."
Heart failure remains a leading cause of hospitalization in the elderly. It accounts for about 300,000 deaths each year in the United States. Heart failure occurs when a person's heart is too weak to properly pump and circulate blood throughout their body.
Dr. Hajjar is already on a path toward approval from the Food and Drug Administration to test the novel SUMO-1 gene therapy in heart failure patients. When it begins, the clinical trial will be the second gene therapy treatment designed to reverse heart failure launched by Dr. Hajjar and his Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai.
The first trial, named CUPID, is in its final phases of testing SERCA2 gene therapy. Phase 1 and phase 2a trial results were positive, demonstrating substantial improvement in clinical events.
In that trial, a gene known as SERCA2 is delivered via an inert virus -- a modified virus without infectious particles. SERCA2 is a gene that produces an enzyme critical to the proper pumping of calcium out of cells. In heart failure, SERCA2 is dysfunctional, forcing the heart to work harder and in the process, to grow larger.
The virus carrying SERCA2 is delivered through the coronary arteries into the heart during a cardiac catheterization procedure. Studies show only a one-time gene therapy dose is needed to restore healthy SERCA2a gene production of its beneficial enzyme. But previous research by Mount Sinai discovered SERCA2 is not the only enzyme that is missing in action in heart failure. A study published in Nature in 2011 by Dr. Hajjar and his research group showed that the SUMO-1 gene is also decreased in failing human hearts. But SUMO-1 regulates SERCA2a's activity, suggesting that it can enhance the function of SERCA2a without altering its levels. A follow-up study in a mouse model of heart failure demonstrated that SUMO-1 gene therapy substantially improved cardiac function.
This new study tested delivery of SUMO-1 gene therapy alone, SERCA2 gene therapy alone, and a combination of SUMO-1 and SERCA2.
In large animal models of heart failure, the researchers found that gene therapy delivery of high dose SUMO-1 alone, as well as SUMO-1 and SERCA2 together, result in stronger heart contractions, better blood flow, and reduced heart volumes, compared to just SERCA2 gene therapy alone.
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Cancer Gene Therapy
A new and better way to treat cancer!
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The new 'Crispr' technique has been hailed as a game-changer by experts It allows scientists to edit DNA with more accuracy than ever before This could allow them to treat genetic disorders like Down syndrome It could also be used to correct gene defects in human embryos
By Ben Spencer
PUBLISHED: 20:32 EST, 6 November 2013 | UPDATED: 20:33 EST, 6 November 2013
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'Game-changer': The Crispr technique for altering DNA is believed to be so accurate it could help cure diseases like HIV, and also save IVF babies from chronic conditions
Leading geneticists last night hailed a jaw-dropping new form of gene therapy which could transform the treatment of incurable disorders.
The new technique allows scientists to edit genetic information with great precision for the first time.
The breakthrough means detailed alterations can be made to human DNA, potentially allowing scientists to treat genetic disorders such as sickle-cell anaemia, Down syndrome and Huntingtons disease.
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DUBLIN--(BUSINESS WIRE)--
Research and Markets (http://www.researchandmarkets.com/research/lvpjrs/gene_therapy) has announced the addition of a new report "Global Gene Therapy Market Report - Analysis of Technologies, Markets and Companies" to their offering.
Genes and DNA are now being introduced without the use of vectors and various techniques are being used to modify the function of genes in vivo without gene transfer. If one adds to this the cell therapy particularly with use of genetically modified cells, the scope of gene therapy becomes much broader.
Gene therapy can now combined with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy report written by him during the past decade including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic
Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described.
Benefits of this report
Who should read this report?
Key Topics Covered:
Executive Summary
1. Introduction
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A modified version of the virus that causes AIDS could be the unlikely saviour of a promising treatment for a host of deadly diseases
IN TECHNOLOGY, it is called the hype cycle: what initially seems a promising breakthrough leads to inflated expectations until it becomes clear that a great deal of time, money and effort will be needed to realise that promise. Disillusionment sets in until the first real successes are reported, and then the hype is on again.
So it has gone with gene therapy. When, in the late 1980s, the genes for debilitating inherited diseases began to be identified, many believed that cures were within reach, by replacing the faulty genes with working ones. But getting the right gene into the right place without doing more harm than good proved tricky. Now, 23 years after the first gene therapy trial for a rare immune disease called ADA-SCID, researchers finally have some successes to report (see "'Bubble kid' success puts gene therapy back on track").
Still, a major barrier remains: cost. The first gene therapy drug to be approved for clinical use, to treat a pancreatic disease, is also the world's most expensive drug. At the moment, the production of modified viruses the vectors used to shuttle genes into a person's cells is prohibitively expensive, meaning only a handful of those with the diseases in question can be treated.
Pharmaceutical companies may have the means and know-how to scale up production, but inherited genetic diseases are not common. So the industry has been reluctant to invest in treatments for them, preferring instead to channel cash towards bigger killers like cancer.
By a stroke of fortune, a promising form of cancer treatment relying on immunotherapy uses the same viral vector that gene therapists are working on to treat diseases like SCID: a modified version of the virus that causes HIV. Some 700 trials using this kind of safer vector are under way, treating a range of degenerative and immune disorders.
It may seem ironic that a virus that has killed so many holds the potential to yield a cure for a host of other deadly diseases, but such is scientific progress: it comes from unexpected places. That should give fresh grounds for the pharma industry to look again at gene therapy. With a bit of ingenuity and effort, gene therapy might finally live up to the hype.
Correction: When this article was first published on 30 October 2013, the strap and standfirst confused HIV and AIDS.
This article appeared in print under the headline "Live up to the hype"
If you would like to reuse any content from New Scientist, either in print or online, please contact the syndication department first for permission. New Scientist does not own rights to photos, but there are a variety of licensing options available for use of articles and graphics we own the copyright to.
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STAMFORD, Conn. -- Stamford's Alliance for Cancer Gene Therapy's "Achieving Cancer Remission with Cell and Gene Therapies" event attracted more than 100 people to New York City last week.
More than 100 donors, scientists, biotech representatives and physicians attended the Tuesday night event at the Harvard Club of New York City, according to a news release.The event "highlighted recent tremendous strides made in combating cancer with cell and gene therapy treatments, and served as appreciation for donors who have committed time and funds to furthering research and clinical trials across the nation," according to the release.
Our donors have allowed top scientific minds to explore this new and promising avenue of cancer treatment, and their philanthropy is directly linked to the lives saved so far, said Barbara Netter, who co-founded the alliance in 2001 with her husband, Edward, in the release.
Netter later said that "much additional research needs to be funded in order to achieve the goal of the fully successful treatment of all types of cancer," according to the release. Netter has assumed the mantle of president of ACGT to "chart a strategic course for the organizations continued success" and further the goal, according to the release.
Guests at the evening event were treated to a reception at the Harvard Club, followed by a salutation from host Dr. Savio Woo, according to the release.
"Dr. Woo Chairman of ACGTs Scientific Advisory Council and Professor of Hematology and Oncology at the Tisch Cancer Institute at Mount Sinai School of Medicine in New York City was instrumental in ACGTs founding over a decade ago," representatives said in the release.
Connie Burnett-West, a cancer survivor "who overcame a critical case lung cancer with gene and cell therapy treatment," also attended the event, according to the release.
Surgery and radiation werent options, and I was told I had limited hope for recovery, Burnett-West said in the release. But after a sixth-month course of gene therapy, Ive been in remission for over 10 years. I could not have imagined a treatment so easy and effective.
The evening also featured a presentation from three of ACGTs Research Fellows, including Carl H. June (M.D., University of Pennsylvania), Laurence Cooper (M.D., Ph.D., MD Anderson Cancer Center) and Michel Sadelain (M.D., Ph.D., Memorial Sloan-Kettering Cancer Center), according to the release. The three "spoke of the breakthroughs and growing momentum that gene and cell therapy has achieved with the support of ACGT," according to the release.
ACGT has the potential to provide less expensive and less harrowing cancer treatment and, ultimately, a cure, Dr. Carl June said in the release. And all of ACGTs life-saving work was funded through philanthropy.
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Stamford's Alliance For Cancer Gene Therapy Celebrates In NYC
Dimension Therapeutics wants to develop a lifetime fix for hemophilia using gene therapy.
On Thursday, another gene therapy startup announced its launch. Dimension Therapeutics hopes to develop virus-delivered gene treatments for rare diseases and its first target is the blood-clotting disorder hemophilia.
The announcement comes just a week after the launch of another gene therapy startup, Spark Therapeutics (see New Gene Therapy Company Launches). One reason that the dashed hopes of gene therapy seem to be mending is that researchers have improved the technologies for delivering genetic fixes. Functional copies of genes are carried by modified viruses, or vectors, into the cells of patients who have missing or dysfunctional copies of those genes. Many groups use vectors based on adeno-associated viruses, or AAVs, which live in most of our bodies already to no ill effect.
Dimension has licensed AAV technology from Washington, D.C.,-based Regenx Biosciences, a company founded by gene therapy pioneer James Wilson. Wilson headed the University of Pennsylvania institute that oversaw a gene therapy trial in 1999 that ended with the death of Jesse Gelsinger, an 18-year-old trial volunteer (see The Glimmering Promise of Gene Therapy). Gelsingers death was blamed on an immune reaction to the experimental therapys viral vector.
That trial used a different kind of virus and since its tragic end, Wilson had searched for better vectors, which he found in AAVs. According to Wired, Wilsons original AAV, AAV1, was the basis for the first gene therapy to be approved in a Western market (see Gene Therapy on the Mend as Treatment Gets Western Approval). Spark Therapeutics is also using a type of AAV to deliver its treatments.
Wilson and his team have since discovered and developed hundreds of modified AAVs, which can target different organs in the body but have been stripped of their ability to replicate. Regenx licensed several vectors to Dimension. A release announcing Dimensions launch suggests that it was Regenx technology that inspired confidence from venture capital firm Fidelity Biosciences to fund the new company:
A core challenge for gene therapy has been the development of safe, efficient vectors to enable delivery of the replacement gene to the correct cells and tissues of the patient to yield benefit, said Fidelity partner and interim CEO of Dimension Thomas Beck. We believe Regenex [vectors] are the most promising approach for in vivo gene therapy.
An early-stage trial of a Regenx vector carrying the gene missing from certain hemophilia patients showed it could correct the disorder (four of the six trial participants were able to quit taking their prophylactic clotting medication) with few side-effects, reported researchers in 2011. The modified virus vectors can still attract the attention of the immune system, but the medical researchers were able to control the immune reaction with immunosuppressive drugs.
While many gene therapy researchers and companies use AAV technology, there are some exceptions. Bluebird Bio, for instance, uses an attenuated version of an HIV viruses that cannot replicate. Bluebird is recruiting patients for alate-stage trial of a gene therapy for a hereditary form of childhood neurodegeneration.
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By Chris Reidy/Globe Staff/October 31, 2013
Fidelity Biosciences, a venture capital firm that is a subsidiary of the parent company of Fidelity Investments, and REGENX Biosciences announced the formation of Dimension Therapeutics, a Cambridge-based gene therapy company focused on developing novel treatments for rare diseases such as hemophilia.
Dimension has completed an undisclosed Series A financing led by Fidelity Biosciences.
In conjunction with its launch, Dimension has entered into an exclusive license and collaboration with REGENX. Through that arrangement, Dimension has acquired preferred access toNAVvector technology and rights in REGENX product programs in multiple rare disease indications.
Gene therapy is a fundamental method of disease intervention, changing a patients genetic code to treat genetic disease, and in some cases providing a potential lifelong benefit following a single treatment, Thomas R. Beck, MD, executive partner at Fidelity Biosciences and interim chief executive of Dimension Therapeutics, said in a statement. A core challenge for gene therapy has been the development of safe, efficient vectors to enable delivery of the replacement gene to the correct cells and tissues of the patient to yield benefit. We believe REGENXNAVvectors are the most promising approach forin vivogene therapy and represent the potential for transformative therapy for patients.
Copyright 2013 Globe Newspaper Company.
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Fidelity Biosciences helps launch company focused on gene therapy products
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Five children with a genetic disease that wipes out their immune system have successfully been treated with gene therapy
Editorial: "Gene therapy needs a hero to live up to the hype"
MOST parents dream of a 5-week-old baby who sleeps through the night, but Aga Warnell knew something was wrong. Her baby, Nina, just wasn't hungry in the way her other daughters had been.
Within weeks, Nina became very ill, says her father, Graeme. She was admitted to hospital with a rotavirus infection. Then she picked up pneumonia.
It turned out Nina had a condition called severe combined immunodeficiency (SCID). She had been born without an immune system due to a genetic defect. It is also known as "bubble boy" disease, since people affected have to live in a sterile environment. "The doctors said 'you need to prepare yourself for the fact that Nina probably isn't going to survive'," says Graeme.
A year-and-a-half later, Nina is a happy little girl with a functioning immune system. She has gene therapy and its latest improvements to thank for it.
SCID was the first condition to be treated with gene therapy more than 20 years ago. A virus was used to replace a faulty gene with a healthy one. But in subsequent trials, four young patients were diagnosed with leukaemia two years after receiving a similar treatment. An 18-year-old also died following a reaction to a virus used in gene therapy for a liver condition. It was the start of a rocky road (see "Trials and tribulations of gene therapy").
Gene therapy has come a long way since, and Nina's case, along with others, mark a turning point: researchers seem to have found a safer way of manipulating our genes.
Preliminary results for the first two children to receive the improved SCID gene therapy 18 months ago were presented at the European Society of Gene and Cell Therapy conference in Madrid, Spain, last week. The children's immune systems have continued to improve since receiving the treatment, says Bobby Gaspar of Great Ormond Street Hospital in London, who led the trial.
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