Review of the Application of RNA Interference Technology in the Pharmaceutical Industry

Toxicologic Pathology

Volumn 35, Issue 3, 2007, Pages 327-336

  Martineau, Henny M ^a   Pyrah, Ian T ^b  

^a SCOTTISH AGRICULTURAL COLLEGE   (United Kingdom)

^b AMGEN INC   (United States)

Author keywords

biotechnology; discovery pathology; drug development; mechanisms of toxicity; molecular pathology; RNA interference

Indexed keywords

DOUBLE STRANDED RNA; MESSENGER RNA; MICRORNA; NAKED SMALL INTERFERING RNA; PIWI INTERACTING RNA; SHORT HAIRPIN RNA; SMALL INTERFERING RNA; SMALL MODULATORY RNA; UNCLASSIFIED DRUG;

ARTHRITIS; BIOTECHNOLOGY; CHEMICAL MODIFICATION; CYTOPLASM; DNA VECTOR; DRUG INDUSTRY; DRUG POTENCY; GENE DELIVERY SYSTEM; GENE SILENCING; GENETIC TRANSFECTION; HISTOPATHOLOGY; HUMAN; INFLUENZA; LIVER DISEASE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PAIN; PRIORITY JOURNAL; PROTEIN SYNTHESIS INHIBITION; REVIEW; RNA DEGRADATION; RNA INTERFERENCE; RNA VECTOR; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; DRUG INDUSTRY; HUMANS; RNA INTERFERENCE; TECHNOLOGY;

EID: 33947398864     PISSN: 01926233     EISSN: None     Source Type: Journal    
DOI: 10.1080/01926230701197107     Document Type: Article

References (82)

1. Ambion Technotes 2006 Retrieved June 7, 2006 from 〈http://www.ambion.com/techlib/detailresults.html〉
2. Berns K Hijmans EM Mullenders J Brummelkamp TR Velds A Heimerikx M Kerkhoven RM Madiredjo M Nijkamp W Weigelt B Agami R Cavet G Linsley PS Beijersbergen RL Bernards R A large-scale RNAi screen in human cells identifies new components of the p53 pathway Nature 2004 428 431 7
3. Bitko V Musiyenko A Shulyayeva O Barik S Inhibition of respiratory viruses by nasally administered siRNA Nat Med 2005 11 50 5
4. Brummelkamp TR Bernards R Agami R A system for stable expression of short interfering RNAs in Mammalian cells Science 2002 296 550 3
5. Budker V Zhang G Danko I Williams P Wolff J The efficient expression of intravascularly delivered DNA in rat muscle Gene Ther 1998 5 272 6
6. Caplen N RNAi quashes polyQ Nat Med 2004 10 775 6
7. Carmell MA Zhang L Conklin DS Hannon GJ Rosenquist TA Germline transmission of RNAi in mice Nat Struct Biol 2003 10 91 2
8. Carrington JC Ambros V Role of MicroRNAs in plant and animal development Science 2003 301 336 8
9. Carthew RW A new RNA dimension to genome control Science 2006 313 305 6
10. Chen C Li L Lodish HF Bartel DP MicroRNAs modulate haematopoeitic lineage differentiation Science 2004 303 83 6
11. Chiu Y Rana TM RNAi in human cells: Basic structural and functional features of small interfering RNA Mol Cell 2002 10 549 61
12. Couzin J Small RNAs make big splash Science 2002 298 2296 7
13. Cristofaro P Ramratnam B RNAi tackles a sexually transmitted disease Nat Bio 2006 24 48 9
14. Cullen BR Derivation and function of small interfering RNAs and microRNAs Virus Res 2004 102 3 9
15. Czauderna F Fechtner M Dames S Aygun H Klippel A Pronk GJ Giese K Kaufmann J Structural variations and stabilizing modifications of synthetic siRNAs in mammalian cells Nucleic Acids Res 2003 31 2705 16
16. Dalby B Cates S Harris A Ohki EC Tilkins ML Price PJ Ciccarone VC Advanced transfection with Lipofectamine 2000 reagent: primary neurons, siRNA and high throughput applications Methods 2004 33 95 103
17. Dass CR Lipoplex-mediated delivery of nucleic acids: factors affecting in vivo transfection J Mol Med 2004 82 1 27
18. Devi GR siRNA-based approaches in cancer therapy Cancer Gene Therapy 2006 1 11 Advance online publication
19. Ding A Li H Lu R Li F Li W RNA silencing: a conserved antiviral immunity of plants and animals Science 2004 296 109 15
20. Dorn G Patel S Wotherspoon G Hemmings-Mieszczak M Barclay J Natt FJC Bevan S Fox A Ganju P Wishart W Hall J siRNA relieves chronic neuropathic pain Nucleic Acids Res 2004 32 e49
21. Downward J RNA interference-based functional genomics in cancer research — an introduction Oncogene 2004 23 8334 5
22. Du Q Thonberg H Wang J Wahlestedt C Liang Z A systematic analysis of the silencing effects of an active siRNA at all single-nucleotide mismatched target sites Nucleic Acids Res 2005 33 1671 7
23. Dykxhoorn DM Lieberman J The silent revolution: RNA interference as basic biology, research tool and therapeutic Ann Rev Med 2005 56 401 23
24. Dykxhoorn DM Novina CD Sharp PA Killing the messenger: short RNAs that silence gene expression Nature Rev 2003 4 457 67
25. 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 411 494 8
26. Feinberg EH Hunter CP Transport of dsRNA into cells by the transmembrane protein SID-1 Science 2003 301 1545 7
27. Fire A Xu S Montgomery M Kostas S Driver S Mello C Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans Nature 1998 391 806 11
28. Ge Q Filip L Bai A Nguyen T Eisen HN Chen J Inhibition of influenza virus production in virus infected mice by RNA interference PNAS 2004 101 8676 81
29. Hagstrom JE Hegge J Zhang G Noble M Budker V Lewis D Herweijer H Wolff J A facile nonviral method for delivering genes and siRNAs to skeletal muscle of Mammalian limbs Mol Therapy 2004 10 1 13
30. Hamar P Song E Kokeny G Chen A Ouyang N Lieberman J Small interfering RNA targeting Fas protects mice against renal ischaemia-reperfusion injury Proc Natl Acad Sci 2004 101 14883 8
31. Harborth J Elbashir SM Vandenburgh K Manninga H Scaringe SA Weber K Tuschl T Sequence, chemical and structural variation of small interfering RNAs and short hairpin RNAs and the effect on Mammalian gene silencing Antisense Nucl Acid Drug Develop 2003 13 83 105
32. Heidel JD Hu S Fang Liu X Triche TJ Davis ME Lack of interferon response in animals to naked siRNAs Nat Biotechnol 2004 22 1579 82
33. Hodges BL Scheule RK Hydrodynamic delivery of DNA Exp Opin Biol Ther 2003 3 911 8
34. Hommel JD Sears RM Georgescu D Simmons DL DiLeone RJ Local gene knockdown in the brain using viral-mediated RNA interference Nat Med 2003 9 1539 43
35. Hutvagner G Zamore PD A micro RNA in a multiple-turnover RNAi enzyme complex Science 2002 297 2056 60
36. Jackson AL Bartz SR Schelter J Kobayashi SV Burchard J Mao M Li B Cavet G Linsley P Expression profiling reveals off-target gene regulation by RNAi Nat Biotechnol 2003 21 635 7
37. Kakizawa Y Furukawa S Kataoka K Block copolymer-coated calcium phosphate nanoparticles sensing intracellular environment for oligodeoxynucleotide and siRNA delivery J Control Rel 2004 97 345 56
38. Ketting RF Plasterk RHA A genetic link between co-supression and RNA interference in C. elegans Nature 2000 404 296 8
39. Kuwabara T Hsieh J Nakashima K Taira K Gage FH A small modulatory dsRNA specifies the fate of adult neural stem Cells Cell 2004 116 779 93
40. Lavery KS King TH Antisense and RNAi: powerful tools in drug target discovery and validation Curr Opin Drug Discov Develop 2003 4 561 9
41. Layzer JM McCaffrey AP Tanner AK Huang Z Kay MA Syllenger BA In vivo activity of nuclease-resistant siRNAs RNA 2004 10 766 71
42. Lewis DL Hagstrom JE Loomis AG Wolff JA Herweijer H Efficient delivery of siRNA for inhibition of gene expression in postnatal mice Nat Gen 2002 32 107 8
43. Li H Xiang W Ding SW Induction and suppression of RNA silencing by an animal virus Science 2002 296 1319 21
44. Lingor P Michel U Scholl U Bahr M Kugler S Transfection of “naked” siRNA results in endosomal uptake and metabolic impairment in cultured neurons Biochem Biophys Res Commun 2004 315 1126 33
45. Liu J Carmell MA Rivas FV Marsden CG Thomson JM Song J Hammond SM Joshua-Tor L Hannon GJ Argonaute 2 is the catalytic engine of mammalian RNAi Science 2004 305 1437 41
46. Liu F Song Y Liu D Hydrodynamics-based transfection in animals by systemic administration for plasmid DNA Gene Ther 1999 6 1258 66
47. Lu PY Xie FY Woodle MC siRNA-mediated antitumorigenesis for drug target validation and therapeutics Curr Opin Mol Therap 2003 5 225 34
48. McCaffrey AP Meuse L Pham TT Conklin DS Hannon GJ Kay MA RNA interference in adult mice Nature 2002 418 38 9
49. McCaffrey AP Nakai H Pandey K Huang Z Salazar F Xu H Wieland SF Marion PL Kay MA Inhibition of hepatitis B virus in mice by RNA interference Nat Biotechnol 2003 21 639 44
50. Medema RH Optimizing RNA interference for application in mammalian cells Biochem J 2004 380 593 603
51. Minakuchi Y Takeshita F Kosaka N Sasaki H Yamamoto Y Kuono M Honma K Nagahara S Hanai K Sano A Kato T Terada M Ochiya T Atelocollagen-mediated synthetic small interfering RNA delivery for effective gene silencing in vitro and in vivo Nucl Acids Res 2004 32 109
52. Mittal V Improving the efficiency of RNA interference in mammals Nat Rev Gen 2004 5 355 65
53. Muratovska A Eccles MR Conjugate for efficient delivery of short interfering RNA into mammalian cells Fed Euro Biochem Soc Lett 2004 558 63 8
54. Naito Y Yamada T Ui-Tei K Morishita S Saigo K siDirect: highly effective, target-specific siRNA design software for mammalian RNA interference Nucl Acids Res 2004 32 W124 9
55. Napoli C Lemieux C Jorgensen R Introduction of a chimeric chalcone synthase gene into petunia results in reversible co-suppression of homologous genes in trans Plant Cell 1990 2 279 89
56. Novina CD Sharp PA The RNAi revolution Nature 2004 430 161 4
57. Palliser D Chowdhury D Wang Q Lee SJ Bronson RT Knipe DM Lieberman J An siRNA-based microbicide protects mice from lethal Herpes simplex virus 2 infection Nature 2006 439 89 94
58. Plasterk RHA RNA silencing: the genome’s immune system Science 2002 20 1263 5
59. Reynolds A Leake D Boese Q Scaringe S Marshall WS Khvorova A Rational siRNA design for RNA interference Nat Biotechnol 2004 22 326 30
60. Rubinson DA Dillon CP Kwiatkowski AV Sievers C Yang L Kopinja J Zhang M McManus MT Gertler FB Scott ML Parijs LV A lentivirus-based system to functionally silence genes in primary mammalian cells, stem cells and transgenic mice by RNA interference Nat Gen 2003 33 401 6
61. Schiffelers RM Xu J Storm G Woodle MC Scaria PV Effects of treatment with small interfering RNA on joint inflammation in mice with collagen-induced arthritis Arthrit Rheum 2005 52 1314 8
62. Schwarz DS Hutvagner G Du T Xu Z Aronin N Samore PD Asymmetry in the assembly of the RNAi enzyme complex Cell 2003 115 199 208
63. Sioud M Sorensen DR Cationic liposome-mediated delivery of siRNAs in adult mice Biochem Biophys Res Commun 2003 312 1220 5
64. Snove O Jr Holen T Many commonly used siRNAs risk off-target activity Biochem Biophys Res Commun 2004 319 256 63
65. Song E Lee S Wang J Ince N Ouyang N Min J Chen J Shankar P Lieberman J RNA interference targeting Fas protects mice from fulminant hepatitis Nat Med 2003 9 347 51
66. Song YK Liu F Zhang G Liu D Hydrodynamics-based transfection: simple and efficient method for introducing and expressing transgenes in animals by intravenous injection of DNA Methods 2002 346 92 105
67. Song J Smith SK Hannon GJ Joshua-Tor L Crystal structure of Argonaute and its implication for RISC slicer activity Science 2004 305 1434 7
68. Sorensen DR Leirdal M Sioud M Gene silencing by systemic delivery of synthetic siRNAs in adult mice J Mol Biol 2003 327 761 6
69. 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 Rohl I Toudjarska I Want G Wuschko S Bumcrot D Koteliansky V Limmer S Manoharan M Vornlocher H Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs Nature 2004 432 173 8
70. Templeton NS Liposomal delivery of nucleic acids in vivo DNA Cell Biol 2002 21 857 67
71. Tomar RS Matta H Chaudhary PM Use of adeno-associated viral vector for delivery of small interfering RNA Oncogene 2003 22 5712 5
72. Tompkins SM Lo C Tumpey TM Epstein SL Protection against lethal influenza virus challenge by RNA interference in vivo Proc Natl Acad Sci 2004 101 8682 6
73. Tousignant JD Gates AL Ingram LA Johnson CL Nietupski JB Cheng SH Eastman SJ Scheule RK Comprehensive analysis of the acute toxicities induced by systemic administration of cationic lipid:plasmid DNA complexes in mice Human Gene Ther 2000 11 2493 513
74. Voinnet O Induction and suppression of RNA silencing: insights from viral infections Nat Rev Gene 2005 6 206 20
75. Wolff JA Malone RW Williams P Chong W Acsadi G Jani A Felgner PL Direct gene transfer into mouse muscle in vivo Science 1990 247 1465 8
76. Xia H Mao Q Eliason S Harper SQ Martins IH Orr HT Paulson HL Yang L Kotin RM Davidson BL RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia Nat Med 2004 10 816 20
77. Yamada T Morishita S Accelerated off-target search algorithm for siRNA Bioinformatics 2004 21 1 10
78. Zamore PD Ancient pathways programmed by small RNAs Science 2002 296 1265 9
79. Zender L Hutker S Liedtke C Tillmann HL Zender S Mundt B Waltemathe M Gosling T Flemming P Malek NP Trautwein C Manns MP Kuhnel F Kubicka S Caspase 8 small interfering RNA prevents acute liver failure in mice Proc Natl Acad Sci 2003 100 7797 802
80. Zhang G Gao X Song YK Vollmer R Stolz DB Gasiorowski JZ Dean DA Liu D Hydroporation as the mechanism of hydrodynamic delivery Gene Ther 2004 11 675 82
81. Zhang G Budker V Wolff JA High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA Human Gene Ther 1999 10 1735 7
82. Zhang Y Bryant J Charles A Boado RJ Pardridge WM Intravenous RNA interference gene therapy targeting the human epidermal growth factor receptor prolongs survival in intracranial brain cancer Clin Cancer Res 2004 10 3667 77