AAV8-TBG-shOctn2

AAV8-TBG-shOctn2 is an experimental gene therapy vector designed for the liver-specific silencing of the Organic Cation/Carnitine Transporter 2 (OCTN2), encoded by the *Slc22a5* gene. It utilizes an adeno-associated virus serotype 8 (AAV8) capsid, which exhibits high tropism for hepatocytes, and a thyroxine-binding globulin (TBG) promoter to restrict the expression of the encoded short hairpin RNA (shRNA) to the liver. The shRNA targets Octn2 mRNA for degradation via the RNA interference (RNAi) pathway. In preclinical models of metabolic dysfunction-associated steatohepatitis (MASH) and hepatocellular carcinoma (HCC), OCTN2 has been shown to promote disease progression by facilitating L-carnitine accumulation, which buffers acetyl groups and disrupts the acetylation of tumor suppressors like p53 and immune-related proteins like MHC-I. By knocking down OCTN2, this vector aims to restore normal metabolic and epigenetic signaling, inhibit tumor growth, and sensitize tumors to immunotherapy.

Other names

AAV8-TBG-shSlc22a5AAV-8-TBG-shSlc22a5AAV 8-TBG-shSlc22a5