Spatial and temporal resolution of the photoreceptors rescue dynamics after treatment with voretigene neparvovec

BACKGROUND: Voretigene neparvovec is a gene therapeutic agent for treatment of retinal dystrophies caused by bi-allelic RPE65 mutations. In this study, we report on a novel and objective evaluation of a retinotopic photoreceptor rescue. METHODS: Seven eyes of five patients (14, 21, 23, 24, 36 years,...

Descripción completa

Detalles Bibliográficos
Autores principales: Stingl, Krunoslav, Kempf, Melanie, Bartz-Schmidt, Karl U, Dimopoulos, Spyridon, Reichel, Felix, Jung, Ronja, Kelbsch, Carina, Kohl, Susanne, Kortüm, Friederike Charlotte, Nasser, Fadi, Peters, Tobias, Wilhelm, Barbara, Wissinger, Bernd, Wozar, Fabian, Zrenner, Eberhart, Fischer, M Dominik, Stingl, Katarina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2022
Materias:
Clinical Science
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9132865/
https://www.ncbi.nlm.nih.gov/pubmed/33472769
http://dx.doi.org/10.1136/bjophthalmol-2020-318286

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9132865/
https://www.ncbi.nlm.nih.gov/pubmed/33472769
http://dx.doi.org/10.1136/bjophthalmol-2020-318286

Ejemplares similares