The Status of RPE65 Gene Therapy Trials: Safety and Efficacy.

Cold Spring Harb Perspect Med

Department of Ophthalmology, Center for Advanced Retinal and Ophthalmic Therapeutics, F.M. Kirby Center for Molecular Ophthalmology, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania 19104.

Published: January 2015

Several groups have reported the results of clinical trials of gene augmentation therapy for Leber congenital amaurosis (LCA) because of mutations in the RPE65 gene. These studies have used subretinal injection of adeno-associated virus (AAV) vectors to deliver the human RPE65 cDNA to the retinal pigment epithelial (RPE) cells of the treated eyes. In all of the studies reported to date, this approach has been shown to be both safe and effective. The successful clinical trials of gene augmentation therapy for retinal degeneration caused by mutations in the RPE65 gene sets the stage for broad application of gene therapy to treat retinal degenerative disorders.

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Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4561397PMC
http://dx.doi.org/10.1101/cshperspect.a017285DOI Listing

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