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Clonal Population of Cells Detected in a Clinical Human Gene Transfer Trial Using Lentiviral Vector.
Posted on:
23 June 2009, source: Recombinant DNA Advisory Committee
The National Institutes of Health Office of Biotechnology Activities (OBA) has been informed that a "relative clonal dominance" was detected during follow-up of a subject who is participating in a French human gene transfer trial being conducted for individuals with β-Thalassemia Major and Sickle Cell Anemia. The clinical trial, sponsored by Genetix France, used hematopoietic stem cells transduced by a self inactivating (SIN) HIV-1 lentiviral vector containing the gene for β-globin under the control of the β-globin promoter. The subject received the gene modified cells in June 2007.
This clonal dominance appears to result from the integration of the vector in the gene encoding for the HMGA2 protein, which is associated with both benign and malignant tumors. The clone however has been stable for five months and the subject remains in good health. In fact, although the subject required almost monthly blood transfusions during the 11 months prior to the gene transfer intervention, the subject has not since required a blood transfusion.
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ASGT Embraces Cell Therapy with Name Change.
Posted on:
29 May 2009, source: ASGCT
The American Society of Gene Therapy (ASGT) will officially change its name Saturday to the American Society of Gene & Cell Therapy (ASGCT), a change intended to better reflect the intimate relationship between the two fields. “This new name more accurately reflects who we are and what we do,” said David M. Bodine, PhD, ASGT president, who led the initiative to add cell therapy to the name. “Gene therapy and cell therapy cannot be separated; each is an integral part of the other.” Added Ken Cornetta, MD, incoming ASGCT president: “Gene therapists are cell therapists. We have the same goal, which is improved patient care.” The change goes into effect at 7:45 a.m. Saturday, May 30, during the business meeting at the ASGT 12th Annual Meeting.
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Moving Gene Therapy Forward With Mobile DNA.
Posted on:
11 May 2009, source: ScienceDaily
Gene therapy is the introduction of genetic material into a patient's cells resulting in a cure or a therapeutic effect. In recent years, it has been shown that gene therapy is a promising technology to treat or even cure several fatal diseases for which there is no attractive alternative therapy. Despite the overall progress, there is still a need to develop improved and safer approaches to deliver genes into cells.
Marinee Chuah, Thierry VandenDriessche, Eyayu Belay and their fellow VIB researchers at K.U. Leuven in collaboration with Zsuzsanna Iszvak and Zoltan Ivics and colleagues at the Max Delbrück Center in Berlin (Germany) have now developed a new non-viral approach that overcomes some of the limitations associated with viral vectors.
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Analysis: Gene therapy has immense potential.
Posted on:
14 April 2009, source: Times Online
After almost two decades, gene therapies have recently started to deliver on their immense promise. Gene therapies have been a part of medicine for almost two decades. Only recently have they started to deliver on their immense promise after a string of setbacks raised doubts about the safety of manipulating human DNA, centre, to treat disease.
The idea behind gene therapy is that by inserting new copies of a particular gene into human cells, it should be possible to correct defects, or to enhance beneficial biological processes. This can be done with viruses, which introduce their own genetic material into the cells they infect, and can be engineered to carry a human gene.
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Gene Therapy Gets Closer to a 'Cure'.
Posted on: 3 March 2009, source: Boston.com
Two decades ago, medicine seemed on the cusp of a revolution. Doctors would soon treat diseases at their very roots, inserting "good" genes to replace patients' faulty ones. Gene therapy was a seductively straightforward idea that offered promise for treating everything from cancer to sickle cell disease.
But only now, after overcoming unexpected scientific obstacles and the high-profile death of a teenage patient, is gene therapy racking up some clear-cut successes. Promising studies are sending ripples of excitement through the field. Some researchers are daring to use the word cure.
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Ark Therapeutics Files Cerepro® Application for European Marketing Authorisation.
Posted on: 8 January 2009, source: Biospace
Ark Therapeutics announced that the Marketing Authorisation Application (MAA) for Cerepro®, Ark’s novel gene-based therapy for operable malignant glioma (brain cancer) which was recently filed with the EMEA, has cleared the validation stage. The Cerepro® MAA application now commences formal review via the centralised procedure which is the standard route for all advanced therapies.
Cerepro® was originally filed for marketing approval in late 2005 and was reviewed by the EMEA with reliance on Phase II data. The review established that more clinical data were needed to confirm the findings and to demonstrate the reproducibility of the results in a larger multi-centre clinical trial. The Phase III study of Cerepro®, completed in July 2007, showed that treatment with Cerepro® resulted in a significant therapeutic benefit, supporting the results of the Phase II clinical studies.
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Proposed ASGT name change to the American Society of Gene and Cell Therapy (ASGCT).
Posted on: 27 November 2008, source: ASGT website / Molecular Therapy
A proposal to change the name of the American Society of Gene Therapy (ASGT) to the American Society of Gene and Cell Therapy (ASGCT) has recently been adopted by the 2008 Membership Committee and forwarded to the Board of Directors of ASGT. The Board has unanimously approved the proposal, which is now being forwarded to the general membership of the Society for a final vote in April of 2009. The Membership Committee’s rationale in making this forward-looking proposal is that the concept of gene therapy includes gene-modified cell therapy, and an inclusive name will empower the Society to expand its membership base and help foster further collaborative research to advance the use of genes and cells as medicine to treat disease.
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