In vivo genome editing in mouse restores dystrophin expression in Duchenne muscular dystrophy patient muscle fibers.
Menglong ChenHui ShiShixue GouXiaomin WangLei LiQin JinHan WuHuili ZhangYaqin LiLiang WangHuan LiJinfu LinWenjing GuoZhiwu JiangXiaoyu YangAnding XuYuling ZhuCheng ZhangLiangxue LaiXiaoping LiPublished in: Genome medicine (2021)
We demonstrated that the clinically relevant CRISPR/Cas9 could restore dystrophin in human muscle cells in vivo in the PDX DMD mouse model. This study demonstrated an approach for the application of gene therapy to other genetic diseases.
Keyphrases
- duchenne muscular dystrophy
- crispr cas
- genome editing
- gene therapy
- mouse model
- skeletal muscle
- induced apoptosis
- muscular dystrophy
- endothelial cells
- poor prognosis
- cell cycle arrest
- case report
- induced pluripotent stem cells
- copy number
- gene expression
- oxidative stress
- endoplasmic reticulum stress
- signaling pathway
- binding protein
- pi k akt