AAV9-pMpz-CD::UPRT-Tnfsf9

**AAV9-pMpz-CD::UPRT-Tnfsf9** is an investigational, locally administered AAV9 gene-therapy vector for **glioblastoma multiforme**. It uses a myelin protein zero promoter to preferentially drive transgene expression in scavenging pericytes within the glioblastoma tumor microenvironment. The vector encodes the cytosine deaminase::uracil phosphoribosyltransferase suicide-gene system, which converts co-administered 5-fluorocytosine into diffusible cytotoxic nucleotide metabolites that kill proliferating tumor cells through a bystander effect, together with Tnfsf9 encoding 4-1BB ligand to enhance local cytotoxic T-cell activation. In a syngeneic mouse glioblastoma model, intracranial administration was reported to induce tumor regression and approximately double survival.

Other names

AAV9-pMpz-CD::UPRT-Tnfsf9