Personalized gene editing helped one baby: can it be rolled out widely?
By Heidi Ledford,
Nature
| 10. 31. 2025
In a world first, a bespoke gene-editing therapy benefited one child. Now researchers plan to launch a clinical trial of the approach.
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.
Now, baby KJ Muldoon’s doctors are gearing up to do it all over again, at least five times over. And faster.
The ground-breaking clinical trial, described on 31 October in the American Journal of Human Genetics2, will deploy an offshoot of the CRISPR–Cas9 gene-editing technique called base editing, which allows scientists to make precise, single-letter changes to DNA sequences. The study is expected to begin next year, after its organizers spent months negotiating with US regulators over ways to simplify the convoluted path a gene-editing therapy normally has to take before it can enter trials.
Developing KJ’s treatment was “a pretty hectic and intense six months”, says Kiran Musunuru, a cardiologist at the Perelman School of Medicine at the University of Pennsylvania in Philadelphia, and one of KJ’s doctors. “But...
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