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

BackgroundVoretigene 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.MethodsSeven eyes of five patients (14, 21, 23, 24, 36 years, 1 ma...

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Veröffentlicht in:	British journal of ophthalmology 2022-06, Vol.106 (6), p.831-838
Hauptverfasser:	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
Format:	Artikel
Sprache:	eng
Schlagworte:	Clinical Science diagnostic tests/investigation dystrophy FDA approval Female Females genetics Humans Male Medical diagnosis Mutation Ophthalmology Photoreceptors Retina Retinal Dystrophies - genetics Surgery Tomography Tomography, Optical Coherence treatment other Visual Acuity Visual Field Tests
Online-Zugang:	Volltext
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