Researchers at the Broad Institute and Jackson Laboratory used prime editing in mouse models to correct genetic mutations causing alternating hemiplegia of childhood (AHC), a rare neurodevelopmental disorder characterized by paralysis and seizures. Targeting five ATP1A3 gene mutations accounting for most AHC cases, treatment reduced symptoms and extended survival significantly. This represents the first demonstration of prime editing therapy efficacy for neurological disease in vivo, supporting potential for rare disease gene editing.