In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration

Leber congenital amaurosis (LCA) is the most common cause of inherited retinal degeneration in children. LCA patients with RPE65 mutations show accelerated cone photoreceptor dysfunction and death, resulting in early visual impairment. It is therefore crucial to develop a robust therapy that not onl...

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Detalles Bibliográficos
Autores principales: Choi, Elliot H., Suh, Susie, Foik, Andrzej T., Leinonen, Henri, Newby, Gregory A., Gao, Xin D., Banskota, Samagya, Hoang, Thanh, Du, Samuel W., Dong, Zhiqian, Raguram, Aditya, Kohli, Sajeev, Blackshaw, Seth, Lyon, David C., Liu, David R., Palczewski, Krzysztof
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8983734/
https://www.ncbi.nlm.nih.gov/pubmed/35383196
http://dx.doi.org/10.1038/s41467-022-29490-3

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