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Chemical and structural modifications of RNAi therapeutics.

Authors :
Ku SH
Jo SD
Lee YK
Kim K
Kim SH
Source :
Advanced drug delivery reviews [Adv Drug Deliv Rev] 2016 Sep 01; Vol. 104, pp. 16-28. Date of Electronic Publication: 2015 Nov 05.
Publication Year :
2016

Abstract

Small interfering RNA (siRNA), a 21-23nt double-stranded RNA responsible for post-transcriptional gene silencing, has attracted great interests as promising genomic drugs, due to its strong ability to silence target genes in a sequence-specific manner. Despite high silencing efficiency and on-target specificity, the clinical translation of siRNA has been hindered by its inherent features: poor intracellular delivery, limited blood stability, unpredictable immune responses and unwanted off-targeting effects. To overcome these hindrances, researchers have made various advances to modify siRNA itself and to improve its delivery. In this review paper, first we briefly discuss the innate properties and delivery barriers of siRNA. Then, we describe recent progress in (1) chemically and structurally modified siRNAs to solve their intrinsic problems and (2) siRNA delivery formulations including siRNA conjugates, polymerized siRNA, and nucleic acid-based nanoparticles to improve in vivo delivery.<br /> (Copyright © 2015 Elsevier B.V. All rights reserved.)

Details

Language :
English
ISSN :
1872-8294
Volume :
104
Database :
MEDLINE
Journal :
Advanced drug delivery reviews
Publication Type :
Academic Journal
Accession number :
26549145
Full Text :
https://doi.org/10.1016/j.addr.2015.10.015