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Testing the genome-editing toolkit in cardiomyopathy

  1. Katie Kingwell
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Genome editing has progressed rapidly from discovery to clinical development, while preclinical studies continue to refine the approaches. Now, two papers in Nature Medicine showcase the potential of two such strategies — base editing and CRISPR–Cas9 — for prevention of hypertrophic cardiomyopathy (HCM) in mouse models. The studies also highlight different advantages and challenges for each genome-editing strategy.

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Nature Reviews Drug Discovery 22, 270 (2023)

doi: https://doi.org/10.1038/d41573-023-00046-4

References

  1. Chai, A. C. et al. Base editing correction of hypertrophic cardiomyopathy in human cardiomyocytes and humanized mice. Nat. Med. 29, 401–411 (2023)

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  2. Reichart, D. et al. Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice. Nat. Med. 29, 412–421 (2023)

    PubMed  Google Scholar 

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