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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Publicat a:Genome Med
Autors principals: 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
Idioma:Inglês
Publicat: BioMed Central 2021
Matèries:
Research
Accés en línia: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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