关键词: 大环多胺, 合成, 阳离子脂质, 基因载体

Abstract: The clinical success of gene therapy requires a safe and effective gene delivery system. Cationic lipids have particularly excellent potential for gene delivery applications because of their lesser immunogenic nature, ability to deliver large pieces of DNA, and ease of handling and preparation techniques. In this study, two cyclen-based amphiphilic molecules including steroid moieties were designed and synthesized, and the structures were characterized by 1H NMR, IR and HRMS. Cationic liposomes were prepared by mixing the molecules with DOPE. Agarose gel electrophoresis demonstrated that the liposomes M1 and M2 could condense plasmid DNA at N/P value of 7 and 5, respectively. The size of the formed lipoplexes were measured to be around 160~220 nm, while the zeta-potential of the lipoplexes were found to be +20~40 mV, suggesting that the synthesized cationic lipids might be of great potential as non-viral gene carrier.

Key words: cyclen, synthesis, cationic lipids, gene vector