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CRISPR gene-editing therapies for hypertrophic cardiomyopathy

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

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Fig. 1: CRISPR gene-editing strategies for HCM.

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Authors and Affiliations

  1. Division of Human Genetics, Children’s Hospital of Philadelphia, Philadelphia, PA, USA

    Alanna Strong

  2. The Center for Applied Genomics, Children’s Hospital of Philadelphia, Philadelphia, PA, USA

    Alanna Strong

  3. Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA

    Alanna Strong

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  1. Alanna Strong
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Correspondence to Alanna Strong.

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The author declares no competing interests.

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Strong, A. CRISPR gene-editing therapies for hypertrophic cardiomyopathy. Nat Med 29, 305–306 (2023). https://doi.org/10.1038/s41591-022-02184-5

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