Lipid-based systemic delivery of siRNA

被引：397

作者：

Tseng, Yu-Cheng ^{[1
]}

Mozumdar, Subho ^{[1
,2
]}

Huang, Leaf ^{[1
]}

机构：

[1] Univ N Carolina, Sch Pharm, Div Mol Pharmaceut, Chapel Hill, NC 27599 USA

[2] Univ Delhi, Dept Chem, Delhi 110007, India

来源：

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

关键词：

Lipid; Liposome; Nanoparticles; siRNA; Systemic delivery; Endosome escape; SMALL INTERFERING RNA; INNATE IMMUNE-RESPONSE; ENDOTHELIAL GROWTH-FACTOR; DOUBLE-STRANDED-RNA; INHIBITS TUMOR-GROWTH; HUMAN BREAST-CANCER; VIVO GENE-TRANSFER; IN-VIVO; HIV-1; INFECTION; PLASMID DNA;

D O I：

10.1016/j.addr.2009.03.003

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

RNAi technology has brought a new category of treatments for various diseases including genetic diseases, viral diseases, and cancer. Despite the great versatility of RNAi that can down regulate almost any protein in the cells, the delicate and precise machinery used for silencing is the same. The major challenge indeed for RNAi-based therapy is the delivery system. In this review, we start with the uniqueness and mechanism of RNAi machinery and the utility of RNAi in therapeutics. Then we discuss the challenges in systemic siRNA delivery by dividing them into two categories-kinetic and physical barriers. At the end, we discuss different strategies to overcome these barriers, especially focusing on the step of endosome escape. Toxicity issues and current successful examples for lipid-based delivery are also included in the review. (C) 2009 Elsevier B.V. All rights reserved.

引用

页码：721 / 731

页数：11

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