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Personalized gene editing helped one baby: can it be rolled out widely?

In a world first, a bespoke gene-editing therapy benefited one child. Now researchers plan to launch a clinical trial of the approach.
By
  1. Heidi Ledford

    1. Heidi Ledford reports for Nature from Seattle, Washington.

Late last year, dozens of researchers spanning thousands of miles banded together in a race to save one baby boy’s life. The result was a world first: a cutting-edge gene-editing therapy fashioned for a single person, and produced in a record-breaking six months1.

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Nature 647, 298-299 (2025)

doi: https://doi.org/10.1038/d41586-025-03566-8

References

  1. Musunuru, K. et al. N. Engl. J. Med. 392, 2235–2243 (2025).

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  2. Ahrens-Nicklas, R. C. & Musunuru, K. Am. J. Hum. Genet. https://doi.org/10.1016/j.ajhg.2025.10.006 (2025).

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