596. Incorporation of a Hepatocyte-Specific Compound in Polycationic Liposomes Enhances Gene Transfection in Hep G2 Cells

Abstract

Background: Cationic liposomes generated from poly(cationic lipid) (PCL) and cholesterol (Chol) have been demonstrated to be a very effective formulation for in vivo gene delivery (Liu et al. Gene Therapy 2003; 10: 180-187), and our polycationic liposome-mediated extracellular superoxide dismutase gene delivery prevented hepatotoxin-induced acute liver injury in mice (Wu et al. Hepatology 2004; 40: 195-204). To enhance targeted gene delivery of cationic liposomes to the liver, particularly to hepatocytes, on which exist abundant asialoglycoprotein receptors (ASGP-R) that mediate ligand-specific endocytosis, we conjugated galactose to a derivative of dimyristylbutanediketone (DMBDK). The resulting new galactose-labeled compound is termed as Gal-S12-DMBDOE.