Duchenne muscular dystrophy: CRISPR/Cas9 treatment

• 来源期刊: 细胞研究（英文版）
• 年，卷（期）: 2016,（5）
• 页码范围: 513-514
• 页数: 2页
• 语种: 中文
• DOI: 10.1038/cr.2016.28

摘要

A novel approach to gone correction by genome editing shows great promise as a treatment for Duchenne muscular dystrophy (DMD).CRISPR/Cas9 delivered by adenoassociated virus to a mouse model for DMD demonstrated improvement in function and histology.