Precision Gene Correction via Prime Editing Restores NADPH Oxidase Activity in Patients with p47phox-Deficient Chronic Granulomatous Disease

A Phase 1/2 clinical trial demonstrates that PM359, an autologous prime-edited CD34+ cell therapy, successfully corrects the delGT mutation in NCF1, restoring essential microbicidal function in patients with p47-CGD without the off-target risks associated with traditional CRISPR-Cas9.