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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.
Retinal gene therapy holds a promise in treating different forms of non-inherited and inherited blindness. In 2008, three independent research groups reported that patients with the rare genetic retinal disease Leber's congenital amaurosis had been successfully treated using gene therapy with adeno-associated virus (AAV).
The original issue focused on viral vectors for gene delivery vectors and use of gene therapy treatments for oncology. Current Gene Therapy was among the first gene therapy focused journals to publish on the use of Alphaviruses for gene therapy, [2] oncolytic virotherapy using adenoviral-mediated gene transfer, [3] and the use of Fas Ligand ...
Nucleoside reverse transcription inhibitors like they are used in anti-HIV therapy were associated with a reduced risk of developing atrophic macular degeneration. This is because Alu elements undergo L1 (protein)-mediated reverse transcription in the cytoplasm resulting in DNA synthesis. First clinical trials are being prepared as of January 2021.
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 ...
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 ...
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 ...
Animal testing for gene therapy began in 2007 with a 2009 breakthrough in squirrel monkeys suggesting an imminent gene therapy in humans. While progress in gene therapy for red-green color blindness has slowed since then, successful human trials are currently underway for achromatopsia, a different form of color vision deficiency.
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