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Review
. 2012:7:3971-80.
doi: 10.2147/IJN.S31897. Epub 2012 Jul 26.

Progress on RNAi-based molecular medicines

Jing Chen  1 , Jianping Xie
Affiliations
Review

Progress on RNAi-based molecular medicines

Jing Chen et al. Int J Nanomedicine. 2012.

Abstract

RNA interference (RNAi) is a promising strategy to suppress the expression of disease-relevant genes and induce post-transcriptional gene silencing. Their simplicity and stability endow RNAi with great advantages in molecular medicine. Several RNAi-based drugs are in various stages of clinical investigation. This review summarizes the ongoing research endeavors on RNAi in molecular medicine, delivery systems for RNAi-based drugs, and a compendium of RNAi drugs in different stages of clinical development. Of special interest are RNAi-based drug target discovery and validation, delivery systems for RNAi-based drugs, such as nanoparticles, rabies virus protein-based vehicles, and bacteriophages for RNA packaging.

Keywords: RNA interference; delivery systems; liposome; molecular medicines; nanoparticle.

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Figures

Figure 1
Figure 1
Schematic representation of RNAi. (A and B) Double-stranded RNA is cleaved into siRNA by Dicer. (C and D). siRNA is unzipped into a guide strand and passenger strand. The latter is subsequently degraded. (E–G). The guide strand is incorporated into a RISC and binds to target mRNA, inducing degradation of mRNA. Abbreviation: RISC, RNA-induced silencing complex.
See this image and copyright information in PMC

References

    1. Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature. 1998;391(6669):806–811. - PubMed
    1. Hannon GJ. RNA interference. Nature. 2002;418(6894):244–251. - PubMed
    1. Meister G, Tuschl T. Mechanisms of gene silencing by double-stranded RNA. Nature. 2004;431(7006):343–349. - PubMed
    1. Novina CD, Sharp PA. The RNAi revolution. Nature. 2004;430(6996):161–164. - PubMed
    1. Pushparaj PN, Aarthi JJ, Manikandan J, Kumar SD. siRNA, miRNA, and shRNA: in vivo applications. J Dent Res. 2008;87(11):992–1003. - PubMed

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