Mini-dCas13X–mediated RNA editing restores dystrophin expression in a humanized mouse model of Duchenne muscular dystrophy

Approximately 10% of monogenic diseases are caused by nonsense point mutations that generate premature termination codons (PTCs), resulting in a truncated protein and nonsense-mediated decay of the mutant mRNAs. Here, we demonstrate a mini-dCas13X–mediated RNA adenine base editing (mxABE) strategy t...

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Detalles Bibliográficos
Autores principales: Li, Guoling, Jin, Ming, Li, Zhifang, Xiao, Qingquan, Lin, Jiajia, Yang, Dong, Liu, Yuanhua, Wang, Xing, Xie, Long, Ying, Wenqin, Wang, Haoqiang, Zuo, Erwei, Shi, Linyu, Wang, Ning, Chen, Wanjin, Xu, Chunlong, Yang, Hui
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2023
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9888377/
https://www.ncbi.nlm.nih.gov/pubmed/36512423
http://dx.doi.org/10.1172/JCI162809

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