Nature, Published online: 31 October 2025; doi:10.1038/d41586-025-03566-8
In a world first, a bespoke gene-editing therapy benefitted one child. Now reseachers plan to launch a clinical trial of the approach.
Personalized gene editing helped one baby: can it be rolled out widely?
Base editors can modify a DNA sequence (shown here in a coloured autoradiogram) at specific target sites, allowing disease-causing mutations to be corrected.Credit: Hank Morgan/Science Photo Library
