Brito Luis, Little Steven, Langer Robert, Amiji Mansoor
Department of Pharmaceutical Sciences, Northeastern University, Boston, Massachusetts 02115, USA.
Biomacromolecules. 2008 Apr;9(4):1179-87. doi: 10.1021/bm7011373. Epub 2008 Feb 29.
Safe and effective nonviral gene delivery and transfection in primary human vascular endothelial cells (EC) and smooth muscle cells (SMC) has tremendous potential for cardiovascular diseases such as in the treatment of coronary restenosis. Using a combination of a cationic biodegradable polymer, poly(beta-amino ester) (PBAE), and a cationic phospholipid, 1,2-dioleoyl-3-trimethylammonium propane (DOTAP), we have engineered a lipopolyplex nanovector system that can transfect EC and SMC cells with reasonably high efficiency. For instance, upon addition of 1.0 microg DNA complexed in lipopolyplexes the transfection efficiency in SMC was 20% and in EC was 33%. The results of this study shows that PBAE-DOTAP-plasmid DNA lipopolyplexes are a promising nonviral vector system for gene delivery and transfection in EC and SMC.
在原代人血管内皮细胞（EC）和平滑肌细胞（SMC）中实现安全有效的非病毒基因递送和转染，对于治疗冠状动脉再狭窄等心血管疾病具有巨大潜力。通过将阳离子可生物降解聚合物聚（β-氨基酯）（PBAE）与阳离子磷脂1,2-二油酰基-3-三甲基铵丙烷（DOTAP）相结合，我们构建了一种脂质多聚体纳米载体系统，该系统能够以相当高的效率转染EC和SMC细胞。例如，加入1.0微克与脂质多聚体复合的DNA后，SMC中的转染效率为20%，EC中的转染效率为33%。本研究结果表明，PBAE-DOTAP-质粒DNA脂质多聚体是一种有前景的非病毒载体系统，可用于EC和SMC中的基因递送和转染。
