Article
Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy.
Human gene therapy - 1 Dec 2018
Pennesi Mark E, Weleber Richard G, Yang Paul, Whitebirch Chris, Thean Beverly, Flotte Terence R, Humphries Margaret, Chegarnov Elvira, Beasley Kathleen N, Stout J Timothy, Chulay Jeffrey D
Abstract excerpt
Previously, results at 2 years after subretinal injection of a recombinant adeno-associated virus vector expressing RPE65 (rAAV2-CB-hRPE65) in eight adults and four children with retinal degeneration caused by RPE65 mutations were reported. Now, results at 5 years after treatment in 11 of these subjects are reported. Subjects received a subretinal injection of rAAV2-CB-hRPE65 in the poorer-seeing eye, at either...
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