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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Udgivet i: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
Format: Artigo
Sprog:Inglês
Udgivet: BioMed Central 2021
Fag:
Research
Online adgang: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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