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The Bespoke Gene Therapy Consortium (BGTC) is a research initiative of the US Foundation for the National Institutes of Health. [ 1 ] [ 2 ] The consortium consists of the FDA , NIH , five non-profit organizations, and 10 pharmaceutical companies.
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;
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 ...
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 ...
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 first retinal gene therapy to be approved by the FDA was Voretigene neparvovec in 2017, which treats Leber's congenital amaurosis, a genetic disorder that can lead to blindness. These treatments also use subretinal injections of AAV vector and are therefore foundational to research in gene therapy for color blindness. [2] [3]
The Center for Cell and Gene Therapy conducts research into numerous diseases, including but not limited to pediatric cancers, [5] [6] diabetes, [7] HIV, glioma [8] and cardiovascular disease. The center has laboratory space in both Baylor College of Medicine and Texas Children's Hospital, and clinical units in Texas Children's and Methodist ...