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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,...
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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BMJ Publishing Group
2022
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Materias: | |
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 |
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