Chemical and structural modifications of RNAi therapeutics

Title
Chemical and structural modifications of RNAi therapeutics
Authors
김광명김선화구숙희조성덕Lee, Yeon Kyung
Issue Date
2016-09
Publisher
Advanced drug delivery reviews
Citation
VOL 104-28
Abstract
Small interfering RNA (siRNA), a 21-23 nt 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. (C) 2015 Elsevier B.V. All rights reserved.
URI
http://pubs.kist.re.kr/handle/201004/64965
ISSN
0169-409X
Appears in Collections:
KIST Publication > Article
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