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Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing

Leber congenital amaurosis (LCA), an inherited retinal degeneration, causes severe visual dysfunction in children and adolescents. In patients with LCA, pathogenic variants, such as RPE65, are evident in specific genes, related to the functions of retinal pigment epithelium and photoreceptors. In co...

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Detalles Bibliográficos
Autores principales:	Jo, Dong Hyun, Jang, Hyeon-Ki, Cho, Chang Sik, Han, Jun Hee, Ryu, Gahee, Jung, Youngri, Bae, Sangsu, Kim, Jeong Hun
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2022
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9792702/ https://www.ncbi.nlm.nih.gov/pubmed/36589710 http://dx.doi.org/10.1016/j.omtn.2022.11.021

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