2009, Number 1
<< Back Next >>
Bioquimia 2009; 34 (1)
Therapeutic approach of RNA interference
Nakamura-López Y, Esparza-Aguilar ML, Garrido-Olvera L, Palomar-Olguín VM, Gallardo-Pérez JC
Language: Spanish
References: 63
Page: 26-36
PDF size: 228.03 Kb.
ABSTRACT
The gene silencing via RNA interference (RNAi), which is controlled by small interfering RNAs (siRNAs), is one of the more used experimental techniques of the last years. There is no doubt that the discovery of RNA molecules that can regulate the expression of genes is the most important advance in Biology during last decades. The reason of its success is owing to the fact that it is a conserved biological mechanism which is present in the cells of a great amount of organisms, including the human. This review shows the great potential of RNAi as physiological knock-down in topics such as gene therapy or cancer, where expression of certain genes favors disease progression. Furthermore, discuss the risks derived of siRNAs introduction and delivery in organisms and the importance of RNAi in a clinical frame where it can to inhibit targets susceptible and non susceptible to drugs.
REFERENCES
Hannon GJ. RNA interference. Nature. 2002; 6894: 244-251.
Moss EG. RNA interference: it´s a small RNA world. Curr Biol. 2001; 19: R772-5.
Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature. 1998; 6669: 806-11.
Caplen NJ, Parrish S, Imani F, Fire A, Morgan RA. Specific inhibition of gene expression by small double-stranded RNAs in invertebrate and vertebrate systems. Proc Natl Acad Sci USA. 2001; 17: 9742-7.
Elbashir SM, Harborth J, Lendeckel W, Yalcin A, Weber K, Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature. 2001; 6836: 494-8.
Bosher JM, Labouesse M. RNA interference: genetic wand and genetic watchdog. Nat Cell Biol. 2000; 2: E31-6.
Tong AW, Zhang YA, Nemunaitis J. Small interfering RNA for experimental cancer therapy. Curr Opin Mol Ther. 2005; 2: 114-24.
Yan KS, Yan S, Farooq A, Han A, Zeng L, Zhou MM. Structure and conserved RNA binding of the PAZ domain. Nature. 2003; 426: 468-74.
Carmell MA, Hannon GJ. RNase III enzymes and the initiation of gene silencing. Nat Struct Mol Biol. 2004; 3: 214-218.
Zamore PD, Tuschl T, Sharp PA, Bartel DP. RNAi: double-stranded RNA directs the ATP dependent cleavage of mRNA at 21 to 23 nucleotide intervals. Cell. 2000; 1: 25-33.
Zamore PD. RNA interference: listening to the sound of silence. Nat Struct Biol. 2001; 9: 746-50.
Parker JS, Roe M, Barford D. Crystal structure of a PIWI protein suggests mechanisms for siRNA recognition and slicer activity. EMBO J. 2004; 23: 4727-37.
Juliano RL, Dixit VR, Kang H, Kim TY, Miyamoto Y, Xu D. Epigenetic manipulation of gene expression: a toolkit for cell biologist. J Cell Biol. 2005; 6: 847-57.
Déctor MA, Arias CF. Interferencia por ARN: un sistema de defensa primitivo. Ciencia. 2004; 55: 25-36.
Heidel JD, Hu S, Liu XF, Triche TJ, Davis ME. Lack of interferon response in animals to naked siRNAs. Nat Biotechnol. 2004; 12: 1579-82.
Agrawal N, Dasaradhi PV, Mohmmed A, Malhotra P, Bhatnagar RK, Mukherjee SK. RNA interference: biology, mechanism and applications. Microbiol Mol Biol Rev. 2003; 4: 657-85.
Verdel A, Songtao J, Gerber S, Sugiyama T, Gygi S, Grewal SIS, et al. RNAi-Mediated targeting of heterochromatin by the RITS complex. Science. 2004; 303: 672-6.
Moss EG. MicroRNAs: hidden in the genome. Curr Biol. 2002; 4: R138-40.
Berkhout B, Jeang KT. RISCy business: MicroRNAs, pathogenesis, and viruses. J Biol Chem. 2007; 282: 26641-5.
Bertrand JR, Pottier M, Vekris A, Opolon P, Maksimenko A, Malvy C. Comparison of antisense oligonucleotides and siRNAs in cell culture and in vivo. Biochem Biophys Res Commun. 2002; 296: 1000-4.
Filleur S, Courtin A, Ait-Si-Ali S, Guglielmi J, Merle C, Harel-Bellan A, et al. SiRNA-mediated inhibition of vascular endothelial growth factor severely limits tumor resistance to antiangiogenic thrombospondin-1 and slows tumor vascularization and growth. Cancer Res. 2003; 63: 3919-22.
Reich SJ, Fosnot J, Kuroki A, Tang W, Yang X, Maguire AM, et al. Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in a mouse model. Mol Vis. 2003; 9: 210-6.
Zender L, Hutker S, Liedtke C, Tillmann HL, Zender S, Mundt B, et al. Caspase 8 small interfering RNA prevents acute liver failure in mice. Proc Natl Acad Sci USA. 2003; 100: 7797-802.
Tompkins SM, Lo CY, Tumpey TM, Epstein SL. Protection against lethal influenza virus challenge by RNA interference in vivo. Proc Natl Acad Sci USA. 2004; 101: 8682-6.
Minakuchi Y, Takeshita F, Kosaka N, Sasaki H, Yamamoto Y, Kouno M, et al. Atelocollagen-mediated synthetic small interfering RNA delivery for effective gene silencing in vitro and in vivo. Nucleic Acids Res. 2004; 32: e109.
Bitko V, Musiyenko A, Shulyayeva O, Barik S. Inhibition of respiratory viruses by nasally administered siRNA. Nat Med. 2005; 11: 50-5.
Kim TW, Lee JH, He L, Boyd DA, Hardwick JM, Hung CF, et al. Modification of professional antigen-presenting cells with small interfering RNA in vivo to enhance cancer vaccine potency. Cancer Res. 2005; 65: 309-16.
Golzio M, Mazzolini L, Moller P, Rols MP, Teissié J. Inhibition of gene expression in mice muscle by in vivo electrically mediated siRNA delivery. Gene Ther. 2005; 12: 246-51.
Weinberg MS, Ely A, Barichievy S, Crowther C, Mufamadi S, Carmona S, et al. Specific inhibition of HBV replication in vitro and in vivo with expressed long hairpin RNA. Mol Ther. 2008; 15: 534-41.
Li SD, Chono S, Huang L. Efficient oncogene silencing and metastasis inhibition via systemic delivery of siRNA. Mol Ther. 2008; 16: 942-6.
Franich NR, Fitzsimons HL, Fong DM, Klugmann M, During MJ, Young D. AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington’s disease. Mol Ther. 2008; 16: 947-56.
Takei Y, Nemoto T, Mu P, Fujishima T, Ishimoto T, Hayakawa Y, et al. In vivo silencing of a molecular target by short interfering RNA electroporation: tumor vascularization correlates to delivery efficiency. Mol Cancer Ther. 2008; 7: 211-21.
Layzer JM, McCafrey AP, Tanner AK, Huang Z, Kay MA, Sullenger BA. In vivo activity of nuclease-resistant siRNAs. RNA. 2004; 10: 766-71.
Jackson AL, Linsley PS. Noise amidst the silence: off-target effects of siRNAs? Trends Genet. 2004; 20: 521-4.
Scacheri PC, Rozenblatt-Rosen O, Caplen NJ, Wolfsberg TG, Umavam L, Lee JC, et al. Short interfering RNAs can induce unexpected and divergent changes in the levels of untargeted proteins in mammalian cells. Proc Natl Acad Sci USA. 2004; 101: 1892-7.
Castanotto D, Sakurai K, Lingeman R, Li H, Shively L, Aagaard L, et al. Combinatorial delivery of small interfering RNAs reduces RNAi efficacy by selective incorporation into RISC. Nucleic Acid Res. 2007; 35: 5154-64.
Doench JG, Sharp PA. Specificity of microRNA target selection in translational repression. Genes Dev. 2004; 18: 504-511.
Devroe E, Silver PA. Therapeutic potencial of retroviral RNAi vectors. Expert Opin Biol Ther. 2004; 3: 319-27.
Valdes VJ, Sampieri A, Sepulveda J, Vaca L. Using double-stranded RNA to prevent in vitro and in vivo viral infections by recombinant baculovirus. J Biol Chem. 2003; 21: 19317-24.
Rondinone CM. Minireview: ribonucleic acid interference for the identification of new targets for the treatment of metabolic diseases. Endocrinology. 2006; 6: 2650-6.
Saltiel AR, Kahn CR. Insulin signalling and the regulation of glucose and lipid metabolism. Nature. 2001; 6865: 799-806.
Gomez-Valades AG, Vidal-Alabro A, Molas M, Boada J, Bermudez J, Bartrons R, et al. Overcoming Diabetes-Induced Hyperglycemia through Inhibition of Hepatic Phosphoenolpyruvate Carboxykinase (GTP) with RNAi. Mol Ther. 2006; 13: 401-10.
Burnett JR, Barrett PH. Apolipoprotein B metabolism: tracer kinetics, models, and metabolic studies. Crit Rev Clin Lab Sci. 2002; 2: 89-37.
Brown MS, Goldstein JL. A receptor-mediated pathway for cholesterol homeostasis. Science. 1986; 4746: 34-47.
Farese RV Jr, Ruland SL, Flynn LM, Stokowski RP, Young SG. Knockout of the mouse apolipoprotein B gene results in embryonic lethality in homozygotes and protection against diet-induced hypercholesterolemia in heterozygotes. Proc Natl Acad Sci USA. 1995; 5: 1774-8.
Soutschek J, Akinc A, Bramlage B, Charisse K, Constien R, Donoghue M, et al. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature. 2004; 7014: 173-8.
Jacque JM, Triques K, Stevenson M. Modulation of HIV-1 replication by RNA interference. Nature. 2002; 6896: 435-438.
Surabhi RM, Gaynor RB. RNA interferente directed against viral and cellular targets inhibits human immunodeficiency virus type 1 replication. J Virol. 2002; 24: 12963-73.
Ter-Brake O, Konstantinova P, Ceylan M, Berkhout B. Silencing of HIV-1 with RNA interference: a multiple shRNA approach. Mol Ther. 2006; 14: 883-92.
Chevalier C, Saulnier A, Benureau Y, Fléchet D, Delgrange D, Colbère-Garapin F, et al. Inhibition of hepatitis C virus infection in cell culture by small interfering RNAs. Mol Ther. 2007; 8: 1452-62.
Brummelkamp TR, Bernards R, Agami R. Stable suppression of tumorigenicity by virus-mediated RNA interference. Cancer Cell. 2002; 3: 243-47.
Rye PD, Stigbrand T. Interfering with cancer: a brief outline of advances in RNA interference in oncology. Tumor Biol. 2004; 5-6: 329-36.
Miao GY, Lu QM, Zhang XL. Downregulation of survivin by RNAi inhibits growth of human gastric carcinoma cells. World J Gastroenterol. 2007; 8: 1170-4.
Clayton J. RNA interference: the silent treatment. Nature. 2004; 7008: 599-605.
Dalmary T, Edwards DR. MicroRNAs and the hallmarks of cancer. Oncogene. 2006; 25: 6170-5.
Ralph GS, Radcliffe PA, Day DM, Carthy JM, Leroux MA, Lee DC, et al. Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model. Nat Med. 2005; 4: 429-33.
Xia X, Zhou H, Huang Y, Xu Z. Allele-specific RNAi selectively silences mutant SOD1 and achieves significant therapeutic benefit in vivo. Neurobiol Dis. 2006; 3: 578-86.
Wang YL, Liu W, Wada R, Murata M, Wada K, Kanazawa I. Clinico-pathological rescue of a model mouse of Huntington’s disease by siRNA. Neurosci Res. 2005; 53: 241-9.
Haque NS, Borghesani P, Isacson O. Therapeutic strategies for Huntington’s disease based on a molecular understanding of the disorder. Mol Med Today. 1997; 3: 175-83.
Zimmermann TS, Lee AC, Akinc A, Bramlage B, Bumcrot D, Fedoruk MN, et al. RNAi mediated silencing in non human primates. Nature. 2006; 441: 111-4.
de Fougeroilles A, Novobrantseva T. siRNA and the lung: research tool or therapeutic drug? Curr Opin Pharm. 2008; 8: 280-5.
Bitko V, Musiyyenko A, Shulyayeva O, Barik S. Inhibition of respiratory viruses by nasally administered siRNA. Nature Med. 2005; 11: 50-5.
Maeda I, Kohara Y, Yamamoto M, Sugimoto A. Large-scale analysis of gene function in Caenorhabditis elegans by high through put RNAi. Curr Biol. 2001; 11: 171-6.