AAV-based gene therapy ameliorates neurological deficits in a mouse model of childhood-onset neurodegeneration with cerebellar atrophy (CONDCA)
Mol Ther. 2025 Aug 2:S1525-0016(25)00628-8. doi: 10.1016/j.ymthe.2025.07.055. Online ahead of print. ABSTRACT Childhood-onset neurodegeneration with cerebellar atrophy (CONDCA) is an autosomal recessive pediatric disorder caused …