Kim Sung Wan
Department of Pharmaceutics and Pharmaceutical Chemistry University of Utah, Salt Lake City, Utah 84112, USA.
Adv Genet. 2005;53:263-74.
Polymeric gene delivery systems have been developed to overcome problems caused by viral carriers. They are low cytotoxic, have no size limit, are convenient in handling, of low cost and reproducible. A Terplex gene delivery system consisting of plasmid DNA, low density lipoprotein and hydropholized poly-L-lysine was designed and characterized. The plasmid DNA, when formulated with stearyl PLL and LDL, forms a stable and hydrophobicity/charge-balanced Terplex system of optimal size for efficient cellular uptake. DNA is still intact after the Terplex formation. This information is expected to be utilized for the development of improved transfection vector for in vivo gene therapy. Terplex DNA complex showed significantly longer retention in the vascular space than naked DNA. This system was used in the augmentation of myocardial transfection at an infarction site with the VEGF gene.
为克服病毒载体引发的问题，人们开发了聚合物基因递送系统。它们细胞毒性低，没有尺寸限制，易于操作，成本低且可重复。设计并表征了一种由质粒DNA、低密度脂蛋白和疏水化聚-L-赖氨酸组成的三元基因递送系统。质粒DNA与硬脂酰聚赖氨酸和低密度脂蛋白配制时，会形成一个稳定且疏水性/电荷平衡的三元系统，其大小最适合细胞有效摄取。三元复合物形成后DNA仍保持完整。这一信息有望用于开发改进的体内基因治疗转染载体。三元DNA复合物在血管空间中的保留时间明显长于裸DNA。该系统用于在梗死部位用血管内皮生长因子（VEGF）基因增强心肌转染。
