Gene Therapy with Voretigene Neparvovec Improves Vision and Partially Restores Electrophysiological Function in Pre-School Children with Leber Congenital Amaurosis

Gene Therapy with Voretigene Neparvovec Improves Vision and Partially Restores Electrophysiological Function in Pre-School Children with Leber Congenital Amaurosis

Leber congenital amaurosis caused by mutations in the RPE65 gene belongs to the most severe early-onset hereditary childhood retinopathies naturally progressing to legal blindness. The novel gene therapy voretigene neparvovec is the first approved causative treatment option for this devastating eye...

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Bibliographic Details
Main Authors: Maximilian J. Gerhardt, Claudia S. Priglinger, Günther Rudolph, Karsten Hufendiek, Carsten Framme, Herbert Jägle, Daniel J. Salchow, Andreas Anschütz, Stylianos Michalakis, Siegfried G. Priglinger
Format: Article
Language:English
Published: MDPI AG 2022-12-01
Series:Biomedicines
Subjects:
gene therapy
voretigene neparvovec
RPE65
leber congenital amaurosis
early-onset retinal dystrophy
electrophysiology
Online Access:https://www.mdpi.com/2227-9059/11/1/103

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