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Gene Therapy is a method of treatment by using DNA as a drug to treat disease by delivering therapeutic DNA into a patient's cells. The most common method of gene therapy includes using DNA that encodes a functional, therapeutic gene to replace a mutated gene.
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Scope and Importance
The other method involves 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 hypothesized in 1972, with the authors influencing 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. By January 2014, about 2,000 clinical trials had been conducted or had been approved using a number of techniques for gene therapy.
The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2014-2024. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades.
International Symposium and Workshops
1. Phacilitate: 11th Annual Cell & Gene Therapy Forum, January 26-28, 2015 Washington DC, USA
2. Cell & Gene Therapy Europe, September 29-30, 2015 Barcelona, Spain
3. Cell Therapy Manufacturing & Gene Therapy Congress, December 2-3, 2015 Brussels, Belgium
Relevant Society and Associations
This page will be updated regularly.
This page was last updated on 14th Sep, 2015
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