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New England Journal of Medicine2025The first published evidence that prime editing works in human patients.
Research paper · Clinical
Prime editing for p47phox-deficient chronic granulomatous disease
The first published evidence that prime editing works in human patients.
Simple explanation
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.
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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.
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