A universal strategy for AAV delivery of base editors to correct genetic point mutations in neonatal PKU mice

Base editing tools enabled efficient conversion of C:G or A:T base pairs to T:A or G:C, which are especially powerful for targeting monogenic lesions. However, in vivo correction of disease-causing mutations is still less efficient because of the large size of base editors. Here, we designed a dual...

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Detalles Bibliográficos
Autores principales: Zhou, Lifang, Su, Jing, Long, Jie, Tao, Rui, Tang, Wenling, Qin, Fengming, Liu, Nan, Wang, Yanhong, Jiao, Yaoge, Hu, Yun, Jiang, Lurong, Li, Li, Yang, Yang, Yao, Shaohua
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/PMC8803597/
https://www.ncbi.nlm.nih.gov/pubmed/35141352
http://dx.doi.org/10.1016/j.omtm.2022.01.001

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