Highlights
The PM359 trial represents a landmark in clinical genome editing, demonstrating the first-in-human application of prime editing to correct the NCF1 delGT mutation. Both study participants achieved prompt and stable engraftment of neutrophils and platelets following myeloablative conditioning. Most significantly, the therapy restored NADPH oxidase activity in neutrophils, as measured by dihydrorhodamine (DHR) assays, which was maintained throughout the follow-up period. The safety profile remained consistent with standard busulfan conditioning, with no evidence of therapy-related severe adverse events or genotoxicity.
