CRISPR gene-editing therapies for hypertrophic cardiomyopathy.
Nature medicine, 29(2): 305-306 PMID 36797479 DOI 10.1038/s41591-022-02184-5
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.