Daily briefing: Custom-made gene-editing therapy for children to enter clinical trial
Article Date: 03 November 2025
Article URL: https://www.nature.com/articles/d41586-025-03619-y
Article Image: https://media.nature.com/lw767/magazine-assets/d41586-025-03619-y/d41586-025-03619-y_50978808.jpg
Summary
Researchers are preparing to trial a personalised base-editing therapy — an offshoot of CRISPR–Cas9 — in at least five more children after it saved baby KJ Muldoon last year. The bespoke treatment, tailored to a child’s specific mutation, was produced in a record six months for KJ; teams now aim to shorten that timeline and begin a formal clinical trial next year. The Nature Briefing also summarises other recent finds: a potent antibiotic precursor discovered in soil bacteria, a failed cloud-seeding attempt in Delhi, and dendrochronology work on ancient coffins revealing past climates.
Key Points
- Personalised base editing was used to save baby KJ Muldoon; researchers plan to trial similar bespoke therapies in at least five more children.
- Base editing enables precise single-letter changes to DNA, offering a potentially faster route to patient-specific treatments than traditional approaches.
- The treatment for KJ was manufactured in six months; clinicians say production time could be reduced further.
- The trial is expected to start next year and will test safety and feasibility of custom-made gene edits in paediatric patients.
- Other highlights in the briefing include discovery of a powerful antibiotic intermediate, an unsuccessful cloud-seeding effort in Delhi, and tree-ring studies from ancient coffins illuminating past climates.
Why should I read this?
Want the quick version? This is huge — a bespoke CRISPR-based therapy actually saved a child and now similar custom fixes are heading into a trial. If you care about how gene therapy could become genuinely personalised and rapid, read the summary here to get the essentials without wading through journals.
Author style
Punchy: this isn’t just another lab breakthrough — it’s clinical translation at speed. If you work in biotech, clinical genetics, or health policy, the details matter: timelines, manufacturing hurdles and ethical oversight will shape whether this approach scales.