Efficient correction of Duchenne muscular dystrophy mutations by SpCas9 and dual gRNAs

Xiang, X; Zhao, XY; Pan, XG; Dong, ZY; Yu, JY; Li, SY; Liang, X; Han, P; Qu, KL; Jensen, JB; Farup, J; Wang, F; Petersen, TS; Bolund, L; Teng, HJ; Lin, L; Luo, YL

Luo, YL (corresponding author), BGI Shenzhen, BGI Qingdao, Qingdao Europe Adv Inst Life Sci, Lars Bolund Inst Regenerat Med, Qingdao 266555, Peoples R China.; Lin, L (corresponding author), Aarhus Univ, Dept Biomed, DK-8000 Aarhus, Denmark.; Teng, HJ (corresponding author), Peking Univ Canc Hosp & Inst, Dept Radiat Oncol, Minist Educ Beijing, Key Lab Carcinogenesis & Translat Res, Beijing, Peoples R China.

MOLECULAR THERAPY-NUCLEIC ACIDS, 2021; 24 (): 403

Abstract

CRISPR gene therapy is one promising approach for treatment of Duchenne muscular dystrophy (DMD), which is caused by a large spectrum of mutations in ......

Full Text Link


Links

期刊讨论 | 中国SCI论文 | 期刊主页 | 投稿经验 | 杂志官网 | 投稿链接 | 作者需知 | PMC链接 | Pubmed全文检索