Narainpersad Nicolisha, Singh Moganavelli, Ariatti Mario
Department of Biochemistry, Non-Viral Gene Delivery Laboratory , University of KwaZulu-Natal, Westville Campus, Durban, South Africa.
Nucleosides Nucleotides Nucleic Acids. 2012;31(3):206-23. doi: 10.1080/15257770.2011.649331.
Liver parenchymal cells are an important target for the treatment of several metabolic and viral disorders. Corrective gene delivery for this purpose is an avenue that is receiving increasing attention. In the present study, we report a novel neo glycolipid that may be formulated into cationic liposomes with or without poly(ethylene glycol) decoration. Lipoplexes formed with plasmid DNA are nuclease resistant and are targeted to the human hepatoblastoma cell line HepG2 by selective asialoglycoprotein receptor mediation. Transfection levels achieved by lipoplexes containing the targeting ligand cholesteryl-3β-N-(4-aminophenyl-β-D-galactopyranosyl) carbamate were sixfold greater than those obtained with similar but untargeted lipoplexes.
肝实质细胞是治疗多种代谢性疾病和病毒感染性疾病的重要靶点。为此目的进行的矫正基因递送是一个受到越来越多关注的途径。在本研究中,我们报道了一种新型的新糖脂,它可以被制备成带有或不带有聚乙二醇修饰的阳离子脂质体。与质粒DNA形成的脂质体对核酸酶具有抗性,并通过选择性去唾液酸糖蛋白受体介导靶向人肝癌细胞系HepG2。含有靶向配体胆固醇-3β-N-(4-氨基苯基-β-D-吡喃半乳糖基)氨基甲酸酯的脂质体实现的转染水平比使用类似但无靶向性的脂质体所获得的转染水平高六倍。