简单说明
Two patients with a rare immune disease had their own blood stem cells corrected by prime editing and returned. The corrected cells took hold, and the immune function they had been missing came back.
深入了解
A first-in-human report of PM359, in which prime editing corrects the NCF1 delGT mutation in autologous CD34+ cells. Both participants engrafted promptly after busulfan conditioning, achieved 69 and 83 per cent dihydrorhodamine-positive neutrophils by day 30, and showed durable restoration of NADPH oxidase activity with early clinical benefit.
Sources
- New England Journal of Medicine · 2025
Prime editing for p47phox-deficient chronic granulomatous disease ↗