The Ladders to Cures Accelerator (L2C) at the Broad Institute congratulates Alex Sousa, Markus Terrey, Holt Sakai, David Liu, Cat Lutz and many other talented scientists, who received one of the first L2C accelerator grants to develop prime and base editing strategies that could efficiently correct prevalent ATP1A3 pathogenic variants with minimal off-target editing.

This ground breaking research demonstrates a proof-of-concept precision gene editing therapy for alternating hemiplegia of childhood (AHC), a rare neurodevelopmental disorder typically caused by mutations in the gene ATP1A3.