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Outcomes and Adverse Effects of Voretigene Neparvovec Treatment for Biallelic RPE65-Mediated Inherited Retinal Dystrophies in a Cohort of Patients from a Single Center

Our study evaluated the morphological and functional outcomes, and the side effects, of voretigene neparvovec (VN) gene therapy for RPE65-mediated inherited retinal dystrophies (IRDs) in 12 eyes (six patients) at the Oxford Eye Hospital with a mean follow-up duration of 8.2 (range 1–12) months. All...

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Detalles Bibliográficos
Autores principales:	Kiraly, Peter, Cottriall, Charles L., Taylor, Laura J., Jolly, Jasleen K., Cehajic-Kapetanovic, Jasmina, Yusuf, Imran H., Martinez-Fernandez de la Camara, Cristina, Shanks, Morag, Downes, Susan M., MacLaren, Robert E., Fischer, M. Dominik
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2023
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10605275/ https://www.ncbi.nlm.nih.gov/pubmed/37892166 http://dx.doi.org/10.3390/biom13101484

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