信号响应性基因载体及其应用研究

Signal Response Gene Carriers

获得合适的基因载体是基因治疗取得成功的关键因素之一。由于病毒载体存在安全隐患问题,人们致力于非病毒载体的研究,但其递送效果仍不理想。为了提高其基因递送效率,改善其靶向性及特异性,并调控其在体内及细胞内的动态,研发具有响应机体内外各种物理、化学及生物信号功能的非病毒载体,逐渐形成了一个重要的研究方向,受到学术界与产业界的极大关注。本文从信号的性质及来源上,将非病毒基因载体分为响应外部物理信号和响应内部化学与生物信号;从响应模式上,将其分为响应单一信号和响应多种信号。系统阐述了此类载体的结构特点、作用机理,并对其研究进展和实际应用进行了综合评述。

The gene carrier is one of key factors for gene therapy. Although non-viral gene carriers have advantages in terms of their safety, the low transfection efficiency is still a major drawback for the research and therapeutic applications. In order to enhance the gene delivery efficiency and specific targeting ability, as well as to control their systematic and intracellular distribution, signal responsive non-viral carriers have been investigated and developed intensively, which can response to various physical, chemical, or biological signals. In this review, typical signal responsive gene carriers are introduced and classified into certain types according to the signal number and source. The latest progress of signal responsive carriers including their molecular structures, responding mechanisms, and biomedical applications are introduced in detail.