Volume 30, No 2, Feb 2020

ISSN: 1001-0602 
EISSN: 1748-7838 2018 
impact factor 17.848* 
(Clarivate Analytics, 2019)

Volume 30 Issue 2, February 2020: 99-100

RESEARCH HIGHLIGHTS

New myotonic dystrophy type 1 mouse model

Yunping Lei¹ and Richard H. Finnell ^1,2

¹Center for Precision Environmental Health, Departments of Molecular and Cellular Biology and Medicine, Baylor College of Medicine, Houston, TX 77030, USA and ² Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA
Correspondence: Richard H. Finnell (Richard.Finnell@bcm.edu)

Myotonic dystrophy type 1 (DM1) is a genetic disorder which compromises multiple organs and for which investigators lack a suitable mouse model for mechanistic and 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.

https://doi.org/10.1038/s41422-020-0276-y

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