肝细胞靶向性Asor-PLL-DNA复合物的构建

目的:构建肝细胞靶向性Asor-PLL-DNA复合物。方法:将携带外源基因的质粒与脱唾液酸糖蛋白结合在一起,形成一种可溶性的蛋白-核酸复合物。结果:该复合物能通过脱唾液酸糖蛋白受体介导的内吞作用,以非病毒感染的方式,将外源基因导入肝细胞并得以表达。结论:和传统的重组病毒介导的基因转移方式相比,该复合物具有更高的靶向性、安全性,该系统的建立为以肝细胞为靶组织的外源基因介导的肝脏相关疾病的基因治疗提供实验基础。

Construction of Liver Cell Targeted Asor-PLL-DNA Complex

To construct a soluble protein-DNA complex. Methods: A plasmid DNA was conjugated with asialoorosomucoid. Asialoorosomucoid can bind to its receptor which is unique in liver cells. Results: This complex which containing foreign gene can be delivered into liver cells by receptor mediated endocytosis in an physiological-like way, and after reacted with lysosome, foreign DNA is released and can be translated into protein. Conclusion:Compared with virus-mediated gene transfer, receptor mediated gene transfer is more safety, more targeted. The construction of protein-DNA complex can provide a basis for liver-related gene therapy.