CRISPR gene-editing therapies for hypertrophic cardiomyopathy

Alanna Strong

doi:10.1038/s41591-022-02184-5

CRISPR gene-editing therapies for hypertrophic cardiomyopathy

Nat Med. 2023 Feb;29(2):305-306. doi: 10.1038/s41591-022-02184-5.

Author

Alanna Strong^{1

2

3}

Affiliations

¹ Division of Human Genetics, Children's Hospital of Philadelphia, Philadelphia, PA, USA. strong.alanna@gmail.com.
² The Center for Applied Genomics, Children's Hospital of Philadelphia, Philadelphia, PA, USA. strong.alanna@gmail.com.
³ Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. strong.alanna@gmail.com.

Abstract

Pre-symptomatic gene editing in preclinical models of hypertrophic cardiomyopathy show therapeutic promise; clinical studies are now needed to assess safety and efficacy in humans.

Publication types

Research Support, N.I.H., Extramural
Comment

MeSH terms

CRISPR-Cas Systems
Cardiomyopathy, Hypertrophic*
Clustered Regularly Interspaced Short Palindromic Repeats*
Gene Editing
Humans

Grants and funding

K08 DK128606/DK/NIDDK NIH HHS/United States