2025
Personalised gene editing & regulatory T cells
MedicineMusunuru, Ahrens-Nicklas et al.; MICrONS Consortium; Brunkow, Ramsdell, Sakaguchi (Nobel)
An infant with CPS1 deficiency received a base-editing therapy designed for his single mutation within months — the first bespoke in-body gene edit. MICrONS mapped a cubic millimetre of mouse visual cortex with its activity. The Nobel in Medicine went to the discovery of regulatory T cells and peripheral immune tolerance.
Why it matters: Shows ‘one patient, one drug’ editing is feasible; links brain wiring to function; explains how the immune system avoids attacking the body.
📄 Musunuru K et al. NEJM 392 (2025). MICrONS Consortium. Nature 640 (2025).