Chorioretinal atrophy following voretigene neparvovec despite the presence of fundus autofluorescence

Chorioretinal atrophy following voretigene neparvovec despite the presence of fundus autofluorescence

Abstract Introduction Leber congenital amaurosis (LCA) type 2, due to disease‐causing variants in RPE65, is characterized by severe visual loss in early infancy. Current treatments include voretigene neparvovec‐rzyl (VN) for RPE65‐associated LCA. Herein, we present the long‐term follow‐up of a patie...

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Bibliographic Details
Main Authors: Masha Kolesnikova, Jose Ronaldo Lima de Carvalho Jr., Rait Parmann, Angela H. Kim, Vinit B. Mahajan, Stephen H. Tsang, Janet R. Sparrow
Format: Article
Language:English
Published: Wiley 2022-11-01
Series:Molecular Genetics & Genomic Medicine
Subjects:
chorioretinal atrophy
gene therapy
inherited retinal disease
leber congenital amaurosis
quantitative autofluorescence
voretigene neparvovec‐rzyl
Online Access:https://doi.org/10.1002/mgg3.2038

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