GSE201587
An efficacious and safe rescue of GM3 synthase deficiency in mice by spatially regulated AAV-mediated ST3GAL5 delivery
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. Overall design: Comparative gene expression profiling analysis of RNA-seq data for wildtype mouse livers treated with PBS, or AAV9.empty, or AAV9.CB.EGFP, or AAV9.CB.hST3GAL5, or AAV9.CB.hST3GAL5_E332K or AAV9.Syn1.hST3GAL5.miR122BS vectors