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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Detalles Bibliográficos
Autores principales: Gerhardt, Maximilian J., Priglinger, Claudia S., Rudolph, Günther, Hufendiek, Karsten, Framme, Carsten, Jägle, Herbert, Salchow, Daniel J., Anschütz, Andreas, Michalakis, Stylianos, Priglinger, Siegfried G.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9855623/
https://www.ncbi.nlm.nih.gov/pubmed/36672611
http://dx.doi.org/10.3390/biomedicines11010103

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