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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.

Go deeper

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

Written by The CRISPR Atlas editorial team Last updated Aug 25, 2026

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Technologies

Prime Editing

Diseases

Chronic Granulomatous Disease

Treatments

PM359
Educational information only This page is a reference, not medical advice. Research and regulatory status change; check the last-updated date above and confirm anything important against the primary sources listed.