Researchers at the Broad Institute and Jackson Laboratory demonstrated that prime editing can successfully correct five genetic mutations causing alternating hemiplegia of childhood (AHC), a severe neurological disorder, in mouse models. The editing resulted in significant improvement in symptoms and survival, establishing a proof of concept for precision gene therapy in neurological conditions. The study included in vitro correction in patient-derived cells and in vivo delivery to the brain, highlighting prime editing’s precision and therapeutic potential for untreatable neurodevelopmental diseases.