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한빛사논문
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Adv. Drug Deliv. Rev., Available online 5 November 2015 | doi:10.1016/j.addr.2015.10.015
Chemical and structural modifications of RNAi therapeutics
Abstract
Sook Hee Ku1, Sung Duk Jo1, Yeon Kyung Lee, Kwangmeyung Kim, Sun Hwa Kim*
Center for Theragnosis, Biomedical Research Institute, Korea Institute of Science and Technology (KIST), Seoul 136-791, Republic of Korea
1 The first two authors contributed equally to this study.
*Corresponding author : Sun Hwa Kim
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.
논문정보
- Review Paper
- 2015년 11월 (BRIC 등록일 2015-11-11)
- 국내 연구진
- 바이오·의료융합 > 바이오센싱 및 나노바이오물질
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