New myotonic dystrophy type 1 mouse model

• 来源期刊: 细胞研究（英文版）
• 年，卷（期）: 2020,（2）
• 页码范围: 99-100
• 页数: 2页
• 语种: 英文

摘要

Myotonic dystrophy type 1 (DM1) is a genetic disorder which compromises multiple organs and for which investigators lack a suitable mouse model for mechanistic end potential drug screening studies.Yin and colleagues using the latest genetic editing method,the CRISPR-Cas9 technique,started from newly established androgenic haploid embryonic stem cells to make a triple heterozygous mutant DM1 mouse model,as well as a quadruple heterozygous mutant DM1 mouse model,which faithfully recapitulates the pathological phenotypes of human DM1.