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Prospect of retinal gene therapy following commercialization of voretigene neparvovec-rzyl for retinal dystrophy mediated by RPE65 mutation

16 Feb 2018

Abstract excerpt

Prospect of retinal gene therapy following commercialization of voretigene neparvovec-rzyl for retinal dystrophy mediated by RPE65 mutationThe approval of voretigene neparvovec-rzyl by the US Food and Drug Administration (FDA), in December 2017, marked the beginning of a new era in medicine in which many inherited diseases will be essentially corrected by gene therapy.Voretigene neparvovec-rzyl, with the trade...

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PMID: 29564403
DOI: 10.1016/j.joco.2018.01.006

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