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A series of clinical trials on gene therapy for RPE65-associated LCA led to the approval of voretigene neparvovec-rzyl (Luxturna) by the Food and Drug Administration ... Gene therapy for glaucoma.
Clinical trials are underway, as are efforts to develop personalized medicines for patients with RVCL. The Clayco Foundation supports research efforts to develop treatments and a gene therapy for RVCL, working working closely with physician-scientists at multiple academic institutions including at the University of Pennsylvania and 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).
Gene therapy is currently not a treatment option, however human clinical trials for both choroideremia and Leber's congenital amaurosis (LCA) have produced somewhat promising results. [14] Clinical trials of gene therapy for patients with LCA began in 2008 at three different sites.
The LEI teams investigate all major causes of blindness including cataracts, diabetes related eye disease, glaucoma, retinal degenerations, corneal, and immune-based diseases. More than 70 scientists at the LEI use a range of technologies to develop treatments for blinding diseases, including gene therapy and telemedicine. [6]
The Ophthalmological Clinical Investigation Centre of the Quinze-Vingts National Eye Hospital opened in 2004. [citation needed]The Clinical Investigation Centre covers all eye diseases but is specialised in exploration of retinal pathologies (macular degeneration, hereditary retinal degenerations, diabetic retinopathies, retinal vascular pathologies) and technological innovations.
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 ...
The lab developed a mouse model of XLRS and successfully treated the condition by gene therapy in mouse [3] [4] and rabbit models. [5] Sieving's team, in coordination with the clinical staff at the National Institutes of Health Clinical Center, is conducting a phase 1 clinical trial to test the therapy in XLRS patients. [6]