Nonviral vector-mediated RNA interference: Its gene silencing characteristics and important factors to achieve RNAi-based gene therapy

被引：125

作者：

Takahashi, Yuki ^{[1
]}

Nishikawa, Makiya ^{[1
]}

Takakura, Yoshinobu ^{[1
]}

机构：

[1] Kyoto Univ, Dept Biopharmaceut & Drug Metab, Grad Sch Pharmaceut Sci, Sakyo Ku, Kyoto 6068501, Japan

来源：

ADVANCED DRUG DELIVERY REVIEWS | 2009年 / 61卷 / 09期

关键词：

Short hairpin RNA; In vivo delivery; Duration; Side effect; Hydrodynamic delivery; SHORT HAIRPIN RNA; PLASMID DNA; BETA-CATENIN; IN-VITRO; DENDRITIC CELLS; TUMOR-GROWTH; EXPRESSION; SIRNA; SHRNA; SUPPRESSION;

D O I：

10.1016/j.addr.2009.04.006

中图分类号：

R9 [药学];

学科分类号：

100702 [药剂学];

摘要：

RNA interference (RNAi) is a potent and specific gene silencing event in which small interfering RNA (siRNA) degrades target mRNA. Therefore, RNAi is of potential use as a therapeutic approach for the treatment of a variety of diseases in which aberrant expression of mRNA causes a problem. RNAi can be achieved by delivering siRNA or vectors that transcribe siRNA or short-hairpin RNA (shRNA). The aim of this review is to examine the potential of nonviral vector-mediated RNAi technology in treating diseases. The characteristics of plasmid DNA expressing shRNA were compared with those of siRNA, focusing on the duration of gene silencing, delivery to target cells and target specificity. Recent progresses in prolonging the RNAi effect, improving the delivery to target cells and increasing the specificity of RNAi in vivo are also reviewed. (C) 2009 Elsevier B.V. All rights reserved.

引用

收藏

页码：760 / 766

页数：7

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