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    Agent in gene therapy promising for retinal dystrophy

    Adeno-associated virus fills void to mutations of gene RPE65

    Use of voretigene neparvovec in gene therapy demonstrates positive outcomes to treat a gene mutation in patients with retinal dystrophy.

    Take-home: Use of voretigene neparvovec in gene therapy demonstrates positive outcomes to treat a gene mutation in patients with retinal dystrophy.

    Philadelphia—In a phase I trial, voretigene neparvovec proved successfully in gene therapy administered to the contralateral eye in patients with inherited retinal dystrophy, caused by mutations of the gene RPE65, according to results published in Lancet.1

    Use of a recombinant adeno-associated virus—voretigene neparvovec (Spark Therapeutics)—introduces the wild-type version of the missing gene RPE65 into the diseased cells, said study author Jean Bennett, PhD, the F.M. Kirby Professor of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia. “This virus is incapable of replicating and causing disease. Its sole purpose is to deliver the normal gene,” she said.

    A previous phase I, dose-escalation study with a unilateral subretinal injection of a recombinant adeno-associated virus vector with the RPE65 gene (voretigene neparvovec) in the worst-seeing eye addressed safety and efficacy in patients with inherited retinal dystrophy caused by RPE65 mutations,2 Dr. Bennett said. That work prompted Dr. Bennett and colleagues to analyze the safety of the administration of voretigene neparvovec to the contralateral eye in patients from the phase I study.

    “After the success of the unilateral injections, we hypothesized that functional vision and retinal and visual function could be further improved by delivering (voretigene neparvovec) to the contralateral, originally better-seeing eye,” the study authors wrote.

    Data of study

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