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

Jul 25, 2023

Genome Medicine. 2021 Apr 12
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
Products used in the paper Details Operation
CRISPR AAV vectors CRISPR AAV vectors were generated by PackGene Biotech Co. (Guangzhou, China). Request Quote

Research Field: Muscle

Keywords: Duchenne muscular dystrophy, Gene editing, CRISPR/Cas9, CRISPR/ Cas12a, Patient-derived xenograft model

AAV Serotype: AAV9

Dose: 25 μL, 1.5E + 12 vg

Routes of Administration: via intramuscular injection

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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