siRNA delivery: from basics to therapeutic applications

被引：36

作者：

Musacchio, Tiziana ^{[1
]}

Torchilin, Vladimir P. ^{[1
]}

机构：

[1] Northeastern Univ, Ctr Pharmaceut Biotechnol & Nanomed, Boston, MA 02115 USA

来源：

FRONTIERS IN BIOSCIENCE-LANDMARK | 2013年 / 18卷

关键词：

siRNA; siRNA conjugate; siRNA delivery systems; Bioconjugates; Micelles; Liposomes; Carbonanotubes; Nanogels; Dendrimers; Lipid-based-particles; Polymer-based-particles; Clinical trials; Review; SMALL INTERFERING RNA; POLYELECTROLYTE COMPLEX MICELLES; IN-VIVO DELIVERY; CELL PENETRATING PEPTIDE; INTRACELLULAR DELIVERY; CANCER-CELLS; MEDIATED DELIVERY; GENE-EXPRESSION; VEGF SIRNA; CHEMICAL-MODIFICATION;

D O I：

10.2741/4087

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

The chance to selectively intervene and stop the development of any gene-dependent disease in different organs and pathologies makes siRNA an ideal therapeutic agent. However, serious issues should be addressed before the real therapeutic use of siRNA. The poor pharmacokinetic properties of siRNA, its short half-life, its low in vivo stability, its fast elimination by kidney filtration and its low transfection efficiency complicate the use of siRNA as a therapeutic molecule. In this review, we will describe the latest and most advanced approaches and strategies undertaken to address these limitations and improve siRNA delivery and its gene silencing efficacy as well as the prospects for its therapeutic applications.

引用

页码：58 / 79

页数：22

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