Researchers at the Broad Institute and Jackson Laboratory used prime editing to successfully correct multiple genetic mutations responsible for alternating hemiplegia of childhood (AHC) in mouse models, extending treated animals’ survival and reducing neurological symptoms. Published in Cell, this marks the first demonstrated therapeutic use of prime editing for a neurological disorder in vivo. The study developed scalable strategies to repair the four most common AHC-causing mutations within the ATP1A3 gene, illustrating the potential for precision gene editing to target diverse mutations across rare diseases.