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The California Institute for Regenerative Medicine (CIRM) is a state agency that supports research and education in the fields of stem cell and gene therapiesIt was created in 2004 after 59% of California voters approved California Proposition 71: the Research and Cures Initiative, [1] which allocated $3 billion to fund stem cell research in California.
European Society of Gene & Cell Therapy (ESGCT) formerly European Society of Gene Therapy (ESGT) is a legally registered professional body which emerged from a small working group in 1992 that focused on human gene therapy. The objectives of the ESGT include the following: promote basic and clinical research in gene therapy;
Translational Genomics Research Institute; California. Clear Labs; Genetic Information Research Institute; Joint Genome Institute (U.S. Department of Energy) Salk Institute for Biological Studies; Illinois. Carl R. Woese Institute for Genomic Biology (University of Illinois, Urbana-Champaign) Maine. The Jackson Laboratory; Maryland. Howard ...
In December 2013 the Doheny Eye Institute entered into an exclusive, long-term affiliation agreement with the University of California Los Angeles, [17] forming the "Doheny Eye Center UCLA". Ronald E. Smith had served as Chairman of Ophthalmology at USC from 1995 to 2013, [ 18 ] and left USC to follow Doheny, and became vice chair of the ...
Current Gene Therapy is a peer-reviewed medical journal published by Bentham Science Publishers.The editor-in-chief is Liang Cheng (Harbin Medical University Harbin, China). ). The focus of this journal is pre-clinical or clinical research on gene the
Clinical trials of gene therapy for sickle cell disease were started in 2014. [227] [228] In February LentiGlobin BB305, a gene therapy treatment undergoing clinical trials for treatment of beta thalassemia gained FDA "breakthrough" status after several patients were able to forgo the frequent blood transfusions usually required to treat the ...
Jesse Gelsinger (June 18, 1981 – September 17, 1999) was the first person publicly identified as having died in a clinical trial for gene therapy. Gelsinger suffered from ornithine transcarbamylase deficiency , an X-linked genetic disease of the liver , the symptoms of which include an inability to metabolize ammonia – a byproduct of ...
Human clinical trials are underway at GenSight Biologics (ClinicalTrials.gov # NCT02064569) and the University of Miami (ClinicalTrials.gov # NCT02161380) to examine the safety and efficacy of mitochondrial gene therapy in LHON. In these trials, participants affected by LHON with the G11778A mutation will have a virus expressing the functional ...
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