title: The Status of RPE65 Gene Therapy Trials: Safety and Efficacy.
abstract: 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.
authors: Department of Ophthalmology, Ocular Genomics Institute, Massachusetts Eye and Ear Infirmary and Harvard Medical School, Boston, Massachusetts 02114.
authors: Eric A
authors: EA
authors: Pierce
authors: 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.
authors: Jean
authors: J
authors: Bennett
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D018076:DNA, Complementary / Q000008:administration & dosage / Q000235:genetics; D004195:Disease Models, Animal; D005544:Forecasting; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors / Q000235:genetics; D006801:Humans; D056965:Injections, Intraocular; D057130:Leber Congenital Amaurosis / Q000628:therapy*; D009154:Mutation / Q000235:genetics*; D012162:Retinal Degeneration / Q000628:therapy*; D055213:Retinal Pigment Epithelium / Q000502:physiology; D016896:Treatment Outcome; D014786:Vision Disorders / Q000235:genetics / Q000628:therapy; D019745:cis-trans-Isomerases / Q000235:genetics*; D000099178:Retinoid Isomerohydrolase
chemical_list: D018076:DNA, Complementary; D019745:cis-trans-Isomerases; D000099178:Retinoid Isomerohydrolase
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