Moreover, intrastriatal injection of rAAVrh.10-sulfoglucosamine sulfohydrolase (Sgsh) transduces neurons in the striatum and hippocampus, restores Sgsh expression in these regions, and ameliorates both GM3 accumulation in the injection site and the disease pathology in the adult mucopolysaccharidosis (MPS) IIIA mouse model (Winner et al., 2016; Gray et al., 2019). Here, SGSH is linked to mucopolysaccharidosis type 3A.