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医药载体系统评论综述
影响因子: 2.9 5年影响因子: 3.72 SJR: 0.736 SNIP: 0.551 CiteScore™: 2.43

ISSN 打印: 0743-4863
ISSN 在线: 2162-660X

医药载体系统评论综述

DOI: 10.1615/CritRevTherDrugCarrierSyst.v23.i2.30
pages 137-164

RNA-Interference Effectors and Their Delivery

A. Doody
Department of Biomedical Engineering and the School of Chemical and Biomolecular Engineering, Cornell University, Ithaca, NY USA
D. Putnam
Department of Biomedical Engineering and the School of Chemical and Biomolecular Engineering, Cornell University, Ithaca, NY USA

ABSTRACT

RNA-interference, the mechanism by which the expression of a specific protein can be reduced or eliminated, has emerged as a potential way to treat disease. RNA-interference effectors, such as small interfering RNA and small hairpin RNA, are double-stranded nucleic acid sequences expressly designed to have homology to sections of the target protein's mRNA, and when present in the cytosol trigger hydrolysis of the mRNA through the RNA-interference pathway. Because RNA-interference effectors are macromolecular and typically polyanionic, their efficacy is poor when not accompanied by a delivery vehicle. This review outlines the RNA-interference mechanism and discusses the delivery of RNA-interference effectors, with an emphasis on synthetic polymer-based delivery systems.


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