Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia: A Nonrandomized Controlled Trial

IMPORTANCE: Achromatopsia linked to variations in the CNGA3 gene is associated with day blindness, poor visual acuity, photophobia, and involuntary eye movements owing to lack of cone photoreceptor function. No treatment is currently available. OBJECTIVE: To assess safety and vision outcomes of supp...

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Detalles Bibliográficos
Autores principales: Fischer, M. Dominik, Michalakis, Stylianos, Wilhelm, Barbara, Zobor, Ditta, Muehlfriedel, Regine, Kohl, Susanne, Weisschuh, Nicole, Ochakovski, G. Alex, Klein, Reinhild, Schoen, Christian, Sothilingam, Vithiyanjali, Garcia-Garrido, Marina, Kuehlewein, Laura, Kahle, Nadine, Werner, Annette, Dauletbekov, Daniyar, Paquet-Durand, François, Tsang, Stephen, Martus, Peter, Peters, Tobias, Seeliger, Mathias, Bartz-Schmidt, Karl Ulrich, Ueffing, Marius, Zrenner, Eberhart, Biel, Martin, Wissinger, Bernd
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Medical Association 2020
Materias:
Original Investigation
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7193523/
https://www.ncbi.nlm.nih.gov/pubmed/32352493
http://dx.doi.org/10.1001/jamaophthalmol.2020.1032

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