AAV8-TBG-Octn2

AAV8-TBG-Octn2 is an adeno-associated virus (AAV) serotype 8-based gene therapy vector designed for liver-specific expression of the OCTN2 (SLC22A5) carnitine transporter. It utilizes the thyroxine-binding globulin (TBG) promoter to drive expression primarily in hepatocytes. OCTN2 is a high-affinity, sodium-dependent plasma membrane transporter responsible for the uptake of L-carnitine into cells, which is essential for the transport of long-chain fatty acids into mitochondria for beta-oxidation. This construct is primarily used in preclinical research to treat models of Primary Carnitine Deficiency (PCD) or to investigate the metabolic role of OCTN2 in liver diseases such as metabolic dysfunction-associated steatohepatitis (MASH) and hepatocellular carcinoma (HCC). By restoring or overexpressing OCTN2, the vector enhances cellular carnitine levels and modulates fatty acid metabolism and downstream signaling pathways.

Other names

AAV8-TBG-SLC22A5AAV-8-TBG-SLC22A5AAV 8-TBG-SLC22A5