AHC gene therapy

**AHC gene therapy** is a nonspecific label referring to experimental gene therapy or gene-editing approaches being investigated for **alternating hemiplegia of childhood**, a rare neurodevelopmental disorder caused most often by **ATP1A3** mutations. The best-defined programs described publicly are **preclinical** CNS-directed genome-editing strategies, including **prime editing** and **base editing**, designed to correct pathogenic ATP1A3 variants such as **D801N** and **E815K** in cellular and mouse models. These approaches have used **AAV9-based in vivo delivery** to the brain in animal studies and aim to restore normal ATP1A3 function, thereby rescuing neuronal sodium-potassium pump activity and improving neurologic phenotypes. No specific named, clinical-stage, marketed, or uniquely branded drug corresponding exactly to the term \"AHC gene therapy\" could be established from available sources; the term appears to denote a therapeutic concept or research area rather than a single clearly identified product.

Other names

alternating hemiplegia of childhood gene therapyATP1A3 gene editing therapyATP-1A3 gene editing therapyATP 1A3 gene editing therapy