Designing Chemically Modified Oligonucleotides for Targeted Gene Silencing

被引：499

作者：

Deleavey, Glen F. ^{[1
]}

Damha, Masad J. ^{[1
]}

机构：

[1] McGill Univ, Dept Chem, Montreal, PQ H3A 0B8, Canada

来源：

CHEMISTRY & BIOLOGY | 2012年 / 19卷 / 08期

关键词：

SMALL INTERFERING RNA; MORPHOLINO ANTISENSE OLIGOMERS; INNATE IMMUNE-RESPONSE; PEPTIDE NUCLEIC-ACIDS; TRANSLATION INITIATION; BIOLOGICAL-ACTIVITY; IN-VIVO; NUCLEOSIDE ANALOGS; PASSENGER-STRAND; STRUCTURAL BASIS;

D O I：

10.1016/j.chembiol.2012.07.011

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

070307 [化学生物学]; 071010 [生物化学与分子生物学];

摘要：

Oligonucleotides (ONs), and their chemically modified mimics, are now routinely used in the laboratory as a means to control the expression of fundamentally interesting or therapeutically relevant genes. ONs are also under active investigation in the clinic, with many expressing cautious optimism that at least some ON-based therapies will succeed in the coming years. In this review, we will discuss several classes of ONs used for controlling gene expression, with an emphasis on antisense ONs (AONs), small interfering RNAs (siRNAs), and microRNA-targeting ONs (anti-miRNAs). This review provides a current and detailed account of ON chemical modification strategies for the optimization of biological activity and therapeutic application, while clarifying the biological pathways, chemical properties, benefits, and limitations of oligonucleotide analogs used in nucleic acids research.

引用

页码：937 / 954

页数：18

共 197 条

[1]

Dumbbell-shaped nanocircular RNAs for RNA interference [J].

Abe, Naoko ;

Abe, Hiroshi ;

Ito, Yoshihiro .

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, 2007, 129 (49) :15108-+

[2]

Modulation of thermal stability can enhance the potency of siRNA [J].

Addepalli, Haripriya ;

Meena ;

Peng, Chang G. ;

Wang, Gang ;

Fan, Yupeng ;

Charisse, Klaus ;

Jayaprakash, K. Narayanannair ;

Rajeev, Kallanthottathil G. ;

Pandey, Rajendra K. ;

Lavine, Gary ;

Zhang, Ligang ;

Jahn-Hofmann, Kerstin ;

Hadwiger, Philipp ;

Manoharan, Muthiah ;

Maier, Martin A. .

NUCLEIC ACIDS RESEARCH, 2010, 38 (20) :7320-7331

[3]

Antisense therapeutics: is it as simple as complementary base recognition? [J].

Agrawal, S ;

Kandimalla, ER .

MOLECULAR MEDICINE TODAY, 2000, 6 (02) :72-81

[4]

Chemical Synthesis of Site-Specifically 2′-Azido-Modified RNA and Potential Applications for Bioconjugation and RNA Interference [J].

Aigner, Michaela ;

Hartl, Markus ;

Fauster, Katja ;

Steger, Jessica ;

Bister, Klaus ;

Micura, Ronald .

CHEMBIOCHEM, 2011, 12 (01) :47-51

[5]

CONFORMATIONAL-ANALYSIS OF SUGAR RING IN NUCLEOSIDES AND NUCLEOTIDES - NEW DESCRIPTION USING CONCEPT OF PSEUDOROTATION [J].

ALTONA, C ;

SUNDARALINGAM, M .

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, 1972, 94 (23) :8205-+

[6]

Tolerance for mutations and chemical modifications in a siRNA [J].

Amarzguioui, M ;

Holen, T ;

Babaie, E ;

Prydz, H .

NUCLEIC ACIDS RESEARCH, 2003, 31 (02) :589-595

[7]

Rational design and in vitro and in vivo delivery of Dicer substrate siRNA [J].

Amarzguioui, Mohammed ;

Lundberg, Patric ;

Cantin, Edouard ;

Hagstrom, James ;

Behlke, Mark A. ;

Rossi, John J. .

NATURE PROTOCOLS, 2006, 1 (02) :508-517

[8]

Amarzguioui Mohammed, 2008, V442, P3, DOI 10.1007/978-1-59745-191-8_1

[9]

The evolution of our thinking about microRNAs [J].

Ambros, Victor .

NATURE MEDICINE, 2008, 14 (10) :1036-1040

[10]

Energetically Important C-H•••F-C Pseudohydrogen Bonding in Water: Evidence and Application to Rational Design of Oligonucleotides with High Binding Affinity [J].

Anzahaee, Maryam Yahyaee ;

Watts, Jonathan K. ;

Alla, Nageswara R. ;

Nicholson, Allen W. ;

Damha, Masad J. .

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, 2011, 133 (04) :728-731

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