Adenine base editing-mediated exon skipping restores dystrophin in humanized Duchenne mouse model

Adenine base editing-mediated exon skipping restores dystrophin in humanized Duchenne mouse model

Abstract Duchenne muscular dystrophy (DMD) affecting 1 in 3500–5000 live male newborns is the frequently fatal genetic disease resulted from various mutations in DMD gene encoding dystrophin protein. About 70% of DMD-causing mutations are exon deletion leading to frameshift of open reading frame and...

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Detalles Bibliográficos
Autores principales: Jiajia Lin, Ming Jin, Dong Yang, Zhifang Li, Yu Zhang, Qingquan Xiao, Yin Wang, Yuyang Yu, Xiumei Zhang, Zhurui Shao, Linyu Shi, Shu Zhang, Wan-jin Chen, Ning Wang, Shiwen Wu, Hui Yang, Chunlong Xu, Guoling Li
Formato: Artículo
Lenguaje:English
Publicado: Nature Portfolio 2024-07-01
Colección:Nature Communications
Acceso en línea:https://doi.org/10.1038/s41467-024-50340-x

Internet

https://doi.org/10.1038/s41467-024-50340-x

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