高聚物基因载体的功能化研究

Functional Polymeric Gene Vectors

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、低成本和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4种基本功能———在细胞外保持稳定的功能、进入细胞的功能、逃离溶酶体的功能和进入细胞核的功能,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,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。

Safe and efficient gene delivery system is one of the most critical points for gene therapy. Polymeric nonviral gene carriers with good properties of safety, easy to design and inexpensive cost, have been utilized to deliver therapeutic genes in vivo. However, traditional polymers as gene vectors only have a few functions which can not meet gene therapy requirements, so it is necessary to decorate traditional polymer gene vectors and get an ideal gene delivery system with multifunction. This review focuses on the development and means of the modification of polymeric gene vectors making them have muhifunction, i.e. the ability to maintain stability outside of cell, the ability of cell entrance, endosome escape, and the ability of nuclear membrane traversing. The main challenges remain and the future direction of polymeric vectors research are discussed.