RNAi and small interfering RNAs in human disease therapeutic applications

被引：244

作者：

Lares, Monica R. ^{[1
]}

Rossi, John J. ^{[1
]}

Ouellet, Dominique L. ^{[1
]}

机构：

[1] City Hope Natl Med Ctr, Beckman Inst, Dept Mol & Cellular Biol, Duarte, CA 91010 USA

来源：

TRENDS IN BIOTECHNOLOGY | 2010年 / 28卷 / 11期

关键词：

HIV-1; INFECTION; GENE-THERAPY; RATIONAL DESIGN; HAIRPIN RNAS; SIRNA; DELIVERY; INHIBITION; PROMOTER; VECTORS; EXPRESSION;

D O I：

10.1016/j.tibtech.2010.07.009

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

Small interfering RNAs (siRNAs) have been shown to effectively downregulate gene expression in human cells, giving them potential to eradicate disease. Prospects for clinical applications are discussed in this review, along with an overview of recent history and our current understanding of siRNAs used for therapeutic application in human diseases, such as cancer and viral infections. Over recent years, progress has been made in lipids, ligands, nanoparticles, polymers and viral vectors as delivery agents and for gene-based expression of siRNA to enhance the efficacy and specificity of these methods while at the same time reducing toxicity. It has become apparent that given the recent advances in chemistry and delivery, RNAi will soon prove to be an important and widely used therapeutic modality.

引用

页码：570 / 579

页数：10

共 78 条

[1] Engineering and optimization of the miR-106b cluster for ectopic expression of multiplexed anti-HIV RNAs [J].

Aagaard L.A. ;

Zhang J. ;

von Eije K.J. ;

Li H. ;

Sætrom P. ;

Amarzguioui M. ;

Rossi J.J. .

Gene Therapy, 2008, 15 (23) :1536-1549

[2] Anti-tumor effect of small interfering RNA targeting the androgen receptor in human androgen-independent prostate cancer cells [J].

Azuma, Koji ;

Nakashiro, Koh-ichi ;

Sasaki, Toyokazu ;

Goda, Hiroyuki ;

Onodera, Jun ;

Tanji, Nozomu ;

Yokoyama, Masayoshi ;

Hamakawa, Hiroyuki .

BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, 2010, 391 (01) :1075-1079

[3]

BAOLIANG G, 2009, ANAT REC HOBOKEN, V292, P633

[4] The inhibitory efficacy of RNA POL III-Expressed long hairpin RNAs targeted to untranslated regions of the HIV-1 5′ long terminal repeat [J].

Barichievy, Samantha ;

Saayman, Sheena ;

Von Eije, Karin J. ;

Morris, Kevin V. ;

Arbuthnot, Patrick ;

Weinberg, Marc S. .

OLIGONUCLEOTIDES, 2007, 17 (04) :419-431

[5] MicroRNAs: Target Recognition and Regulatory Functions [J].

Bartel, David P. .

CELL, 2009, 136 (02) :215-233

[6] Prevention of interferon-stimulated gene expression using microRNA-designed hairpins [J].

Bauer, M. ;

Kinkl, N. ;

Meixner, A. ;

Kremmer, E. ;

Riemenschneider, M. ;

Foerstl, H. ;

Gasser, T. ;

Ueffing, M. .

GENE THERAPY, 2009, 16 (01) :142-147

[7] Low-level shRNA Cytotoxicity Can Contribute to MYC-induced Hepatocellular Carcinoma in Adult Mice [J].

Beer, Shelly ;

Bellovin, David I. ;

Lee, Joyce S. ;

Komatsubara, Kimberly ;

Wang, Lora S. ;

Koh, Huishan ;

Boerner, Kathleen ;

Storm, Theresa A. ;

Davis, Corrine R. ;

Kay, Mark A. ;

Felsher, Dean W. ;

Grimm, Dirk .

MOLECULAR THERAPY, 2010, 18 (01) :161-170

[8] Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease [J].

Cavazzana-Calvo, M ;

Hacein-Bey, S ;

Basile, CD ;

Gross, F ;

Yvon, E ;

Nusbaum, P ;

Selz, F ;

Hue, C ;

Certain, S ;

Casanova, JL ;

Bousso, P ;

Le Deist, F ;

Fischer, A .

SCIENCE, 2000, 288 (5466) :669-672

[9] Dual-target gene silencing by using long, synthetic siRNA duplexes without triggering antiviral responses [J].

Chang, Chan Il ;

Kang, Hye Suk ;

Ban, Changill ;

Kim, Soyoun ;

Lee, Dong-ki .

MOLECULES AND CELLS, 2009, 27 (06) :689-695

[10] Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells [J].

Czauderna, F ;

Fechtner, M ;

Dames, S ;

Aygün, H ;

Klippel, A ;

Pronk, GJ ;

Giese, K ;

Kaufmann, J .

NUCLEIC ACIDS RESEARCH, 2003, 31 (11) :2705-2716

← 1 2 3 4 5 6 7 8 →