TY - JOUR
T1 - Chemical and structural modifications of RNAi therapeutics
AU - Ku, Sook Hee
AU - Jo, Sung Duk
AU - Lee, Yeon Kyung
AU - Kim, Kwangmeyung
AU - Kim, Sun Hwa
N1 - Publisher Copyright:
© 2015 Elsevier B.V.
PY - 2016
Y1 - 2016
N2 - 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.
AB - 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.
KW - Chemical modification
KW - RNA interference
KW - Structural modification
KW - siRNA
KW - siRNA delivery
UR - http://www.scopus.com/inward/record.url?scp=84957687437&partnerID=8YFLogxK
U2 - 10.1016/j.addr.2015.10.015
DO - 10.1016/j.addr.2015.10.015
M3 - Review article
C2 - 26549145
AN - SCOPUS:84957687437
SN - 0169-409X
VL - 104
SP - 16
EP - 28
JO - Advanced Drug Delivery Reviews
JF - Advanced Drug Delivery Reviews
ER -