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Abstract

October to December 2020 | Vol. 13 | Issue 4 | 20-21
Gene Therapies for Genetic disorders
Neha Agrawal
Department of Medical Genetics, Sanjay Gandhi Postgraduate Institute of Medical Sciences, Lucknow, India
Address for Correspondence Email: ne4444ha@gmail.com
 
Abstract
RPE65-associated LCA is an inherited retinal degeneration. In 2017, the US Food and Drug Administration (FDA) approved the first directly administered gene therapy that targets RPE65, voretigeneneparvovec-rzyl (Luxturna). Voretigeneneparvovec-rzyl uses the adeno-associated virus (AAV) vector and is delivered as a single subretinal injection. After this the retinal cells start producing the protein. However, cautious optimism is necessary, as the drug is not expected to restore normal vision, and only about half of treated patients had minimally meaningful improvement in short-term studies. Wang et al. reviewed six studies with 82 patients, of which only one was a randomised control trial. They found that gene therapy was effective up to 2 years post treatment in terms of improvement of best-corrected visual acuity and full-field light sensitivity threshold to blue flashes.
 
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