CRISPR gene-editing therapies for hypertrophic cardiomyopathy.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 36797479.
- Also identified by DOI 10.1038/s41591-022-02184-5 and PMC identifier 9969544.
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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.
Medical subject headings
- Clustered Regularly Interspaced Short Palindromic Repeats
- Cardiomyopathy, Hypertrophic