The end result of many retinal dystrophies is photoreceptor cell death and the irreversible loss of vision. To prevent the progressive loss of retinal neurons, and to slow down or reverse the progression of the disease, gene therapies are currently under study to correct or replace the altered genes responsible for different retinopathies. Another therapeutic strategy that is under intensive...
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The patient, treated at the Casey Eye Institute in Portland, Oregon, belongs to a group of 18 people affected with Leber congenital amaurosis. These patients will participate in the BRILLIANCE study, the first clinical trial to determine the safety and efficacy of a therapy based on the CRISPR-Cas9 gene editing technique (AGN-151587, EDIT-101). It is the first example of an...
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This February Moorfields Eye Hospital in London and Radcliffe Hospital in Oxford have started treating patients affected by Leber congenital amaurosis (LCA) with the Luxturna gene therapy. LCA is a childhood-onset severe retinal dystrophy, which causes irreversible blindness for which, until now, no treatment was available. Luxturna (voretigene neparvovec) is also the first gene therapy approved for the treatment of an...
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Researchers at the Johns Hopkins University have tested an alternative route to deliver gene therapy vectors to the retina that could potentially help patients with age-related macular degeneration (AMD) and inherited retinal disorders (IRDs). The new route of administration, called suprachoroidal injection, has been investigated initially in preclinical experiments in animals and uses a microneedle to inject gene-carrying therapeutic viruses...
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