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In vivo genome editing rescues photoreceptor degeneration via a Cas9/RecA-mediated homology-directed repair pathway

Although Cas9-mediated genome editing has been widely used to engineer alleles in animal models of human inherited diseases, very few homology-directed repair (HDR)–based genetic editing systems have been established in postnatal mouse models for effective and lasting phenotypic rescue. Here, we dev...

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Detalles Bibliográficos
Autores principales: Cai, Yuan, Cheng, Tianlin, Yao, Yichuan, Li, Xiao, Ma, Yuqian, Li, Lingyun, Zhao, Huan, Bao, Jin, Zhang, Mei, Qiu, Zilong, Xue, Tian
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6469935/
https://www.ncbi.nlm.nih.gov/pubmed/31001583
http://dx.doi.org/10.1126/sciadv.aav3335