Researchers from the Broad Institute and Jackson Laboratory utilized prime editing—a precision CRISPR-derived gene editing technology—to correct multiple ATP1A3 gene mutations responsible for alternating hemiplegia of childhood (AHC), a severe and rare neurological disorder. In vitro correction of mutations in patient-derived iPSCs achieved up to 90% efficiency. In vivo trials in mouse models demonstrated substantial reduction in neurological symptoms and increased survival, marking the first therapeutic application of prime editing in the brain. This breakthrough offers a scalable blueprint for treating various genetic neurological diseases.