CRISPR vs cholesterol: can gene editing prevent heart disease?

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Promising results from a small clinical trial highlight a growing interest in designing gene-editing treatments for common illnesses.

A CRISPR–Cas9 gene-editing therapy has halved people’s cholesterol levels in a small clinical trial — raising hopes that, with further study, gene editing could one day be harnessed to provide a one-stop treatment for a common cause of heart disease.

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Nature 647, 832-833 (2025)

doi: https://doi.org/10.1038/d41586-025-03711-3

References

  1. Laffin, L. J. et al. New Engl. J. Med. https://doi.org/10.1056/NEJMoa2511778 (2025).

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  2. Lazzarotto, C. R. et al. Preprint at bioRxiv https://doi.org/10.1101/2025.02.10.637517 (2025).

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