非病毒性载体在肝脏疾病基因治疗中的研究进展  

Progress of Non-viral Vectors in Gene Therapy of Hepatic Disease

作　　者：谭平萍[1] 冯德云[1] 

机构地区：[1]中南大学湘雅医学院病理学系,湖南长沙410078

出　　处：《现代生物医学进展》2009年第6期1178-1182,共5页Progress in Modern Biomedicine

基　　金：国家自然科学基金资助项目(No.30671846)

摘　　要：基因治疗肝脏疾病的新策略已引起高度关注,在肝病的基因治疗中,最关键的是如何将治疗基因特异性地导入肝细胞中并适当表达。在过去的二十多年里,受体介导的基因给药系统广泛用于肝靶向基因递送,但一些非病毒载体的基因传递效率不高。本文综述了目前最常用的非病毒载体,包括其理化性质、优点和局限性,基因递送作用机理以及修饰后在肝靶向基因治疗中的应用,并综述了在肝细胞基因传递中常用的电穿孔技术和流体力学注射法等物理方法以及如何实现其最优化的转染率。As a new promising therapeutical strategy for the treatment of liver diseases, gene therapy has drawn people＇s great attention. In gene therapy of hepatic diseases, it is critical to get therapeutic genes into hepatocyte for appropriate expression specifically. Over the past twenty years, receptor mediated gene delivery system have been extensively studied for liver-targeted gene delivery. However, non-viral gene delivery is still inefficient. This article reviewed the current non-viral vector used commonly, including their physicochemical properties, advantages and limitations, mechanisms of action for gene delivery, and applications modified for liver-targeted gene therapy, and the physical methods for hepatocyte gene delivery, such as electroporation and hydrodynamic injection. It also reviewed how to achieve optimal transfection efficiency.

关 键 词：肝靶向 基因治疗 非病毒载体 裸DNA 阳离子脂质体 阳离子聚合物 

分 类 号：R575[医药卫生—消化系统]