Vision rescue via unconstrained in vivo prime editing in degenerating neural retinas

Retinitis pigmentosa (RP) is an inherited retinal dystrophy causing progressive and irreversible loss of retinal photoreceptors. Here, we developed a genome-editing tool characterized by the versatility of prime editors (PEs) and unconstrained PAM requirement of a SpCas9 variant (SpRY), referred to...

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Detalles Bibliográficos
Autores principales: Qin, Huan, Zhang, Wenliang, Zhang, Shiyao, Feng, Yuan, Xu, Weihui, Qi, Jia, Zhang, Qian, Xu, Chunxiu, Liu, Shanshan, Zhang, Jia, Lei, Yushuang, Liu, Wanqin, Feng, Shuyu, Wang, Jingjing, Fu, Xuefei, Xu, Zifen, Li, Ping, Yao, Kai
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Rockefeller University Press 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10037108/
https://www.ncbi.nlm.nih.gov/pubmed/36930174
http://dx.doi.org/10.1084/jem.20220776

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