AAV9 gene therapy ameliorates GNAO1 encephalopathy-associated hyperlocomotion in vivo
Aug. 28, 2024
Mutations in the GNAO1 gene are tied to neurological disorders characterized by movement abnormalities and developmental delay. GNAO1 encodes the protein guanine nucleotide-binding protein G(o) subunit α, which is highly expressed in the brain. Among the mutations, R209H results in dystonia, choreoathetosis and developmental delay without seizures.