In vivo genome editing in mouse restores dystrophin expression in Duchenne muscular dystrophy patient muscle fibers
Brief intro:
- Author: Menglong Chen, Hui Shi, Shixue Gou, Xiaomin Wang, Lei Li, Qin Jin, Han Wu, Huili Zhang, Yaqin Li, Liang Wang, Huan Li, Jinfu Lin, Wenjing Guo, Zhiwu Jiang, Xiaoyu Yang, Anding Xu, Yuling Zhu, Cheng Zhang, Liangxue Lai, and Xiaoping Li
- Journal: Genome Medicine
- Doi: https://www.doi.org/10.1186/s13073-021-00876-0
- Publication Date: 2021 Apr 12
Products/Services used in the paper
Quotation shows PackGene:CRISPR AAV vectors were generated by PackGene Biotech.
Research Field:Muscle
AAV Serotype:AAV9
Targeted organ:DMD muscle fibers
Animal or cell line strain:NSI mice/PDX mouse
Abstract
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 strategy for permanently curing DMD.
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