Generation of a DMD loss-of-function mutant human embryonic stem cell lines by CRISPR base editing

Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive disorder, which is caused mostly by frame-disrupting, out-of-frame variation in the dystrophin (DMD) gene. Loss-of- function mutations are the most common type of mutation in DMD, accounting for approximately 60–90% of all DMD variation...

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Autores principales:	Hui Jin, Hong Fu, Jingjing Wang, Zhongming Wang, Jing Liu, Fengjie Han, Haijun Zheng, Youxu Jiang
Formato:	Artigo
Lenguaje:	Inglês
Publicado:	Elsevier 2024-04-01
Colección:	Stem Cell Research
Acceso en línea:	http://www.sciencedirect.com/science/article/pii/S1873506124000412
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