AAV-mediated base-editing therapy ameliorates the disease phenotypes in a mouse model of retinitis pigmentosa

Base editing technology is an ideal solution for treating pathogenic single-nucleotide variations (SNVs). No gene editing therapy has yet been approved for eye diseases, such as retinitis pigmentosa (RP). Here, we show, in the rd10 mouse model, which carries an SNV identified as an RP-causing mutati...

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Detalles Bibliográficos
Autores principales: Wu, Yidong, Wan, Xiaoling, Zhao, Dongdong, Chen, Xuxu, Wang, Yujie, Tang, Xinxin, Li, Ju, Li, Siwei, Sun, Xiaodong, Bi, Changhao, Zhang, Xueli
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10427680/
https://www.ncbi.nlm.nih.gov/pubmed/37582961
http://dx.doi.org/10.1038/s41467-023-40655-6

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10427680/
https://www.ncbi.nlm.nih.gov/pubmed/37582961
http://dx.doi.org/10.1038/s41467-023-40655-6

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