Gene silencing by chemically modified siRNAs

被引：30

作者：

Engels, Joachim W. ^{[1
]}

机构：

[1] Goethe Univ Frankfurt, Inst Organ Chem & Chem Biol, D-60438 Frankfurt, Germany

来源：

NEW BIOTECHNOLOGY | 2013年 / 30卷 / 03期

关键词：

DOUBLE-STRANDED-RNA; INTERFERING RNAS; MAMMALIAN-CELLS; NUCLEIC-ACID; EXPRESSION; STABILITY; POTENT; THERAPEUTICS; ACTIVATION; ANTISENSE;

D O I：

10.1016/j.nbt.2012.07.002

中图分类号：

Q5 [生物化学];

学科分类号：

071010 ; 081704 ;

摘要：

RNA interference (RNAi) has not only already risen as a gold standard for validating gene function in basic science studies, but also holds great promise as a new therapeutic paradigm. Advantages of RNAi-based therapeutics include relatively fast initial screening and the ability to target proteins not yet addressable by traditional drug design strategies. In this review we describe the development of chemically modified small inhibiting siRNAs and their application as potential therapeutics during the past decade. Focus is on proper siRNA design, choice of chemical modification and how to circumvent immunogenicity as well as off-target effects.

引用

页码：302 / 307

页数：6

共 50 条

[1] Cheminformatics Modeling of Gene Silencing for Both Natural and Chemically Modified siRNAs
Dong, Xialan
Zheng, Weifan
MOLECULES, 2022, 27 (19):
[2] Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
Soutschek, J
Akinc, A
Bramlage, B
Charisse, K
Constien, R
Donoghue, M
Elbashir, S
Geick, A
Hadwiger, P
Harborth, J
John, M
Kesavan, V
Lavine, G
Pandey, RK
Racie, T
Rajeev, KG
Röhl, I
Toudjarska, I
Wang, G
Wuschko, S
Bumcrot, D
Koteliansky, V
Limmer, S
Manoharan, M
Vornlocher, HP
NATURE, 2004, 432 (7014) : 173 - 178
[3] Efficient Gene Silencing in Brain Tumors with Hydrophobically Modified siRNAs
Osborn, Maire F.
Coles, Andrew H.
Golebiowski, Diane
Echeverria, Dimas
Moazami, Michael P.
Watts, Jonathan K.
Sena-Esteves, Miguel
Khvorova, Anastasia
MOLECULAR CANCER THERAPEUTICS, 2018, 17 (06) : 1251 - 1258
[4] Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
Jürgen Soutschek
Akin Akinc
Birgit Bramlage
Klaus Charisse
Rainer Constien
Mary Donoghue
Sayda Elbashir
Anke Geick
Philipp Hadwiger
Jens Harborth
Matthias John
Venkitasamy Kesavan
Gary Lavine
Rajendra K. Pandey
Timothy Racie
Kallanthottathil G. Rajeev
Ingo Röhl
Ivanka Toudjarska
Gang Wang
Silvio Wuschko
David Bumcrot
Victor Koteliansky
Stefan Limmer
Muthiah Manoharan
Hans-Peter Vornlocher
Nature, 2004, 432 : 173 - 178
[5] Silencing of c-myc gene expression using enzymatically and chemically synthesized siRNAs
Kabilova, T. O.
Vladimirova, A. V.
Repkova, M. N.
Ven'yaminova, A. G.
Chernolovskaya, E. L.
Vlasov, V. V.
MOLECULAR BIOLOGY, 2006, 40 (06) : 936 - 944
[6] Silencing of c-myc gene expression using enzymatically and chemically synthesized siRNAs
T. O. Kabilova
A. V. Vladimirova
M. N. Repkova
A. G. Ven’yaminova
E. L. Chernolovskaya
V. V. Vlasov
Molecular Biology, 2006, 40 : 936 - 944
[7] Suppression of MDR1 gene expression by chemically modified siRNAs
Logashenko, E. B.
Vladimirova, A. V.
Volkov, A. A.
Repkova, M. N.
Ven'yaminova, A. G.
Zenkova, M. A.
Chernolovskaya, E. L.
Vlassov, V. V.
RUSSIAN CHEMICAL BULLETIN, 2006, 55 (07) : 1275 - 1283
[8] Suppression of MDR1 gene expression by chemically modified siRNAs
E. B. Logashenko
A. V. Vladimirova
A. A. Volkov
M. N. Repkova
A. G. Ven’yaminova
M. A. Zenkova
E. L. Chernolovskaya
V. V. Vlassov
Russian Chemical Bulletin, 2006, 55 : 1275 - 1283
[9] Therapeutic applications of chemically modified siRNAs
Vargeese, Chandra
ABSTRACTS OF PAPERS OF THE AMERICAN CHEMICAL SOCIETY, 2006, 231
[10] Silencing of genes responsible for polyQ diseases using chemically modified single-stranded siRNAs
Fiszer, Agnieszka
Ellison-Klimontowicz, Marianna E.
Krzyzosiak, Wlodzimierz J.
ACTA BIOCHIMICA POLONICA, 2016, 63 (04) : 759 - 764

← 1 2 3 4 5 →