In vivo genome editing in mouse restores dystrophin expression in Duchenne muscular dystrophy patient muscle fibers

BACKGROUND: Mutations in the DMD gene encoding dystrophin—a critical structural element in muscle cells—cause Duchenne muscular dystrophy (DMD), which is the most common fatal genetic disease. Clustered regularly interspaced short palindromic repeat (CRISPR)-mediated gene editing is a promising stra...

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Detalhes bibliográficos
Publicado no:Genome Med
Main Authors: Chen, Menglong, Shi, Hui, Gou, Shixue, Wang, Xiaomin, Li, Lei, Jin, Qin, Wu, Han, Zhang, Huili, Li, Yaqin, Wang, Liang, Li, Huan, Lin, Jinfu, Guo, Wenjing, Jiang, Zhiwu, Yang, Xiaoyu, Xu, Anding, Zhu, Yuling, Zhang, Cheng, Lai, Liangxue, Li, Xiaoping
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2021
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8042958/
https://ncbi.nlm.nih.gov/pubmed/33845891
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13073-021-00876-0
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