An efficacious and safe rescue of GM3 synthase deficiency in mice by spatially regulated AAV-mediated ST3GAL5 delivery

ABSTRACT: The first-generation rAAV-ST3GAL5 replacement vector employing a ubiquitous promoter led to hepatic injury and death caused by high off-target ST3GAL5 expression when administered systemically. In contract, a seconed-geneeration vector optimized for CNS-restricted expression of ST3GAL5 eliminated liver toxicity while preserving neurotherapeutic effects.

ORGANISM(S): Mus musculus

PROVIDER: GSE201587 | GEO | 2023/01/01

REPOSITORIES: GEO