阳离子聚合物在非病毒基因转染中的研究进展

Advances in cationic polymers used as nonviral vectors for gene delivery

摘要:

基因治疗为治疗先天性遗传疾病和严重后天获得性疾病提供了一条新途径。目前，基因载体分为两类：病毒载体和非病毒载体。病毒载体转染效率高，但由于某些病毒载体存在免疫原性、致癌性、宿主DNA插入整合等缺点，从而限制了它们的应用。非病毒载体具有价格低、制备简单、安全有效、无免疫原性等优点，成为基因载体研究的热点。阳离子多聚物是非病毒载体的典型代表。文中综述近年来阳离子多聚物作为基因载体的研究现状和进展，重点介绍了阳离子多聚物基因载体的分类和与DNA的相互作用和传递机制。

关键词:基因治疗，非病毒载体，阳离子聚合物

Abstract:

Gene therapy has been considered as a promising method for treatment of many diseases, such as acquired and genetic diseases. At present, there are two major vehicles for gene delivery including viral vectors and nonviral vectors. Viral vectors appear as high gene transfection efficiency, but some deficiencies such as inflammatory responses, recombination and mutagenesis have limited their use. On account of low pathogenicity, safety and cost-effectiveness, nonviral vectors have been attracted much attention. Cationic polymers are one of the nonviral vectors which have been widely studied. This review focuses on the structure of the cationic polymers and the interaction mechanism between the vector and DNA. We try to provide a framework for the future design and synthesis of nonviral vectors with high transfection efficiency and low toxicity for gene therapy.

Key words:gene therapy, nonviral vectors, cationic polymer