AAV9-Mediated Intra-Striatal Delivery of GNAO1 Reduces Hyperlocomotion in Gnao1 Heterozygous R209H Mutant Mice [Chemotherapy, Antibiotics, and Gene Therapy]
ASPET
JUNE 12, 2024
Mutations in the GNAO1 gene, which encodes the abundant brain G-protein Gα o , result in neurologic disorders characterized by developmental delay, epilepsy, and movement abnormalities. There are over 50 mutant alleles associated with GNAO1 disorders; the R209H mutation results in dystonia, choreoathetosis, and developmental delay without seizures.
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