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[基因治疗中的药物递送系统]

[Drug delivery systems in gene therapy].

作者信息

Takakura Y

机构信息

Graduate School of Pharmaceutical Sciences, Kyoto University.

出版信息

Nihon Rinsho. 1998 Mar;56(3):691-5.

PMID:9549358
Abstract

The use of nonviral vectors is an attractive in vivo gene delivery strategy that is simpler and lacks some risks inherent in viral systems. Liposomes and receptor-mediated polycation systems are promising carriers for delivery and expression of plasmid DNA encoding genes into the target cells. Many barriers need to be overcome for successful in vivo DNA delivery using these carrier systems. Various factors such as the extent of DNA condensation, particle size of the DNA complex, route of administration, stability against nucleases, target sites, in vivo disposition, binding to cell surface receptor and internalization, intracellular trafficking affect the in vivo gene delivery and expression. This chapter will focus on the current status and perspectives of the plasmid DNA delivery systems for in vivo gene therapy.

摘要

使用非病毒载体是一种有吸引力的体内基因递送策略,它更简单,并且没有病毒系统固有的一些风险。脂质体和受体介导的聚阳离子系统是用于将编码基因的质粒DNA递送并表达至靶细胞的有前景的载体。使用这些载体系统成功进行体内DNA递送需要克服许多障碍。诸如DNA凝聚程度、DNA复合物的颗粒大小、给药途径、对核酸酶的稳定性、靶位点、体内处置、与细胞表面受体的结合及内化、细胞内运输等各种因素都会影响体内基因递送和表达。本章将聚焦于用于体内基因治疗的质粒DNA递送系统的现状与前景。

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