Mouse models for ultra-rare disorder could pave the way for nervous system gene editing therapies
Peer-Reviewed Publication
Updates every hour. Last Updated: 2-Jul-2025 15:10 ET (2-Jul-2025 19:10 GMT/UTC)
JAX scientists have developed a mouse models for AHC, a rare and severe neurological disorder in children, paving the way for gene-editing therapies and deeper understanding of disease progression and treatment.