基于多糖聚合物的非病毒基因载体的研究进展

Advances in Polysaccharide Polymer-based Non-viral Vehicle

基因治疗是通过一定的载体或手段将功能性外源基因导入受体细胞中,通过外源基因的表达产物或是通过外源基因抑制某些基因的转录或翻译,达到治疗的目的。基因载体有2种:病毒载体传递体系和非病毒载体传递体系。病毒载体传递体系因安全问题使其在临床应用中多有限制。非病毒载体有着诸多优势,但较低的转染效率限制了其在研究及临床上的应用。本文综述近年来一类基于多糖聚合物设计的非病毒载体的研究进展,并阐述其在基因治疗中的优势及应用。

Gene therapy is defined as the use of certain vector or approaches to treat diseases by delivering exogenous gene into patient＇s cells, which involves the gene that encodes a therapeutic protein or directly interacts with other biomolecules. The two major methods utilizing vectors are classified as viral and non-viral delivery system. Non-viral delivery system is superior in security compared with viral vector in clinical trial, but is considerably limited to its deficiency in gene delivering. In this review, the advances of polysaccharide polymer-based non-viral vehicle in recent years are presented, as well as its application in already published trails of gene therapy.