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A generative artificial-intelligence tool has designed a synthetic CRISPR system that successfully edits human DNA and sharply reduces off-target effects.

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  1. Pascal Notin

    1. Pascal Notin is in the Department of Systems Biology, Harvard Medical School, Boston, Massachusetts 02115, USA.

Despite emerging clinical successes, current genome editors suffer from off-target effects and can trigger unwanted responses from the immune system, limiting their broader therapeutic applications. Writing in Nature, Ruffolo et al.1 present OpenCRISPR-1, the first AI-generated CRISPR–Cas protein to edit human DNA successfully. The work demonstrates how machine learning can be used to engineer functional biological systems that extend beyond those found in nature.

doi: https://doi.org/10.1038/d41586-025-02135-3

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Competing Interests

The author declares no competing interests.

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