高聚物基因载体的功能化研究

寻找安全、高效的基因载体是基因治疗的关键问题之一，聚合物基因载体具有低毒、低成本、可设计性强等优良的性质，是未来基因载体发展的方向。传统的高聚物作为基因载体时，功能单一，不能完全满足基因治疗的需要，因此对载体进行功能化修饰是十分必要的。本文在分析基因治疗过程的基础上，详述了基因载体应具有的四种基本功能——在细胞外保持稳定的功能、进入细胞的功能、逃离溶酶体的功能、进入细胞核的功能，并详细介绍了有关基因载体功能化修饰的方法和目前国际上的进展情况，最后本文总结了高聚物载体目前存在的主要问题及未来的发展方向。

The Research of Functional Polymeric Gene Vector

Developing of safe、efficient gene delivery systems is one of the most critical points for gene therapy. Polymeric non-viral gene carriers having good properties, such safety、easy to design、inexpensive, have been utilized to deliver therapeutic genes in vivo. However, traditional polymers as gene vector only have a few function can not meet gene therapy requirement, so it is necessary to decorate traditional polymer gene vectors, getting ideal gene vectors with multi-function. This review focuses on the development and means of the modification of polymeric gene vector making them have multi-function—the ability to maintain stability outside of cell、the ability of cell entrance、the ability of endosome escape and the ability of nuclear membrane traversing, at last, we highlight the main challenges that remain and the future direction of polymeric vectors research.