Retinal gene therapy in RPE-65 gene mediated inherited retinal dystrophy

Jalil, A; Ivanova, T; Moussa, G; Parry, NRA; Black, GCM

Jalil, A (通讯作者)，Manchester Royal Eye Hosp, Oxford Rd, Manchester M13 9WL, Lancs, England.

EYE, 2023; 37 (9): 1874

Abstract

Background Voretigene neparvovec (VN) is a gene therapeutic agent for treatment of retinal dystrophies caused by bi-allelic RPE65 mutations. We illust......

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