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LEBER CONGENITAL AMAUROSIS DUE TO CEP290 MUTATIONS—SEVERE VISION IMPAIRMENT WITH A HIGH UNMET MEDICAL NEED: A Review

PURPOSE: Leber congenital amaurosis due to CEP290 mutations (LCA10) is an inherited retinal disease that often results in severe visual impairment or blindness in early childhood. Currently, there are no approved treatments, highlighting the considerable unmet medical need associated with LCA10. We...

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Detalles Bibliográficos
Autores principales:	Leroy, Bart P., Birch, David G., Duncan, Jacque L., Lam, Byron L., Koenekoop, Robert K., Porto, Fernanda B. O., Russell, Stephen R., Girach, Aniz
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Retina 2021
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8078118/ https://www.ncbi.nlm.nih.gov/pubmed/33595255 http://dx.doi.org/10.1097/IAE.0000000000003133

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