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BioWorld - Wednesday, March 4, 2026
Breaking News: BioWorld Science 2025 Year in ReviewSee today's BioWorld Science
Home » AAV9 gene therapy ameliorates GNAO1 encephalopathy-associated hyperlocomotion in vivo
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Genetic/congenital

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.
BioWorld Science Genetic/congenital Neurology/psychiatric Gene therapy

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