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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Following a long-standing CGS tradition, we present a selection of our favorite Biopolitical Times posts of the past year.
In 2025, we published up to four posts every month, written by 12 authors (staff, consultants and allies), some in collaboration and one simply credited to CGS.
These titles are presented in chronological order, except for three In Memoriam notices, which follow. Many more posts that are worth your time can be found in the archive. Scroll down and “VIEW...
