The therapeutic potential of RNA interference

FEBS Letters

Volumn 579, Issue 26, 2005, Pages 5996-6007

  Uprichard, Susan L ^a  

^a SCRIPPS RESEARCH INSTITUTE   (United States)

Author keywords

Delivery; Drug development; Gene suppression; RNA interference; Safety

Indexed keywords

RNA; SMALL INTERFERING RNA; VIRUS VECTOR;

AMYOTROPHIC LATERAL SCLEROSIS; DRUG DELIVERY SYSTEM; DRUG EFFICACY; DRUG POTENCY; DRUG SAFETY; DRUG SENSITIZATION; GENE CONTROL; GENE REPRESSION; GENE SILENCING; GENE TARGETING; HUMAN; PRIORITY JOURNAL; RETINA MACULA AGE RELATED DEGENERATION; RNA INTERFERENCE; SEVERE ACUTE RESPIRATORY SYNDROME; SHORT SURVEY; VIRAL GENE DELIVERY SYSTEM; VIRUS GENE; VIRUS INHIBITION; VIRUS REPLICATION;

ANIMALS; ANTINEOPLASTIC AGENTS; APOPTOSIS; BIOTECHNOLOGY; DRUG DELIVERY SYSTEMS; DRUG INDUSTRY; EYE DISEASES; GENE EXPRESSION REGULATION; GENE EXPRESSION REGULATION, VIRAL; GENETIC TECHNIQUES; HUMANS; INFLAMMATION; RNA INTERFERENCE; RNA, SMALL INTERFERING; RNA, VIRAL;

EID: 26944499005     PISSN: 00145793     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.febslet.2005.08.004     Document Type: Short Survey

References (173)

1. J. Kurreck Antisense technologies. Improvement through novel chemical modifications Eur. J. Biochem. 270 8 2003 1628 1644
2. S.M. Elbashir, J. Harborth, W. Lendeckel, A. Yalcin, K. Weber, and T. Tuschl Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells Nature 411 6836 2001 494 498
3. S.M. Elbashir, W. Lendeckel, and T. Tuschl RNA interference is mediated by 21- and 22-nucleotide RNAs Genes Dev. 15 2 2001 188 200
4. C. Novina, and P. Sharp The RNAi revolution Nature 430 6996 2004 161 164
5. W. Li, and S. Ding Viral suppressors of RNA silencing Curr. Opin. Biotechnol. 12 2 2001 150 154
6. C. Sullivan, and D. Ganem A virus-encoded inhibitor that blocks RNA interference in mammalian cells J. Virol. 79 12 2005 7371 7379
7. Y. Bennasser, S. Le, M. Benkirane, and K. Jeang Evidence that HIV-1 encodes an siRNA and a suppressor of RNA silencing Immunity 22 5 2005 607 619
8. K. Nakahara, and R. Carthew Expanding roles for miRNAs and siRNAs in cell regulation Curr. Opin. Cell. Biol. 15 2 2004 127 133
9. P. Xu, M. Guo, and B. Hay MicroRNAs and the regulation of cell death Trends Genet. 20 12 2004 617 624
10. V. Ambros The functions of animal microRNAs Nature 431 7006 2004 350 353
11. J. Beal Silence is golden: can RNA interference therapeutics deliver? Drug Discov. Today 10 3 2005 169 172
12. T. Brummelkamp, R. Bernards, and R. Agami Stable suppression of tumorigenicity by virus-mediated RNA interference Cancer Cell 2 3 2002 243
13. J. Martinez, A. Patkaniowska, H. Urlaub, R. Luhrmann, and T. Tuschl Single-stranded antisense siRNAs guide target RNA cleavage in RNAi Cell 110 5 2002 563 574
14. H. Ding, D. Schwarz, A. Keene, B. Affarel, L. Fenton, X. Xia, Y. Shi, P. Zamore, and Z. Xu Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis Aging Cell 2 4 2003 209 217
15. V. Miller, H. Xia, G. Marrs, C. Gouvion, G. Lee, B. Davidson, and H. Paulson Allele-specific silencing of dominant disease genes Proc. Natl. Acad. Sci. USA 100 12 2003 7195 7200
16. M. Wilda, U. Fuchs, W. Wossmann, and A. Borkhardt Killing of leukemic cells with a BCR/ABL fusion gene by RNA interference (RNAi) Oncogene 21 37 2002 5716 5724
17. P. Gonzalez-Alegre, V. Miller, B. Davidson, and H. Paulson Toward therapy for DYT1 dystonia: allele-specific silencing of mutant TorsinA Ann. Neurol. 53 6 2003 781 787
18. T. Achenbach, B. Brunner, and K. Heermeier Oligonucleotide-based knockdown technologies: antisense versus RNA interference Chembiochem 4 10 2003 928 935
19. J. Bertrand, M. Pottier, A. Vekris, P. Opolon, A. Maksimenko, and C. Malvy Comparison of antisense oligonucleotides and siRNAs in cell culture and in vivo Biochem. Biophys. Res. Commun. 296 4 2002 1000 1004
20. M. Miyagishi, M. Hayashi, and K. Taira Comparison of the suppressive effects of antisense oligonucleotides and siRNAs directed against the same targets in mammalian cells Antisense Nucleic Acid Drug Dev. 13 1 2003 1 7
21. Y. Xu, H. Zhang, D. Thormeyer, O. Larsson, Q. Du, J. Elmen, C. Wahlestedt, and Z. Liang Effective small interfering RNAs and phosphorothioate antisense DNAs have different preferences for target sites in the luciferase mRNAs Biochem. Biophys. Res. Commun. 306 3 2003 712 717
22. R. Kretschmer-Kazemi Far, and G. Sczakiel The activity of siRNA in mammalian cells is related to structural target accessibility: a comparison with antisense oligonucleotides Nucleic Acids Res. 31 15 2003 4417 4424
23. A. Grunweller, E. Wyszko, B. Bieber, R. Jahnel, V. Erdmann, and J. Kurreck Comparison of different antisense strategies in mammalian cells using locked nucleic acids, 2′-O-methyl RNA, phosphorothioates and small interfering RNA Nucleic Acids Res. 31 12 2003 3185 3193
24. Y. Zhang, M. Taylor, W. Samson, and M. Phillips Antisense inhibition: oligonucleotides, ribozymes, and siRNAs Methods Mol. Med. 106 2005 11 24
25. S. Uprichard, B. Boyd, A. Althage, and F. Chisari Clearance of hepatitis B virus from the liver of transgenic mice by short hairpin RNAs Proc. Natl. Acad. Sci. USA 102 3 2005 773 778
26. J. Harborth, S. Elbashir, K. Vandenburgh, H. Manninga, S. Scaringe, K. Weber, and T. Tuschl Sequence, chemical, and structural variation of small interfering RNAs and short hairpin RNAs and the effect on mammalian gene silencing Antisense Nucleic Acid Drug Dev. 13 2 2003 83 105
27. A. Reynolds, D. Leake, Q. Boese, S. Scaringe, W. Marshall, and A. Khvorova Rational siRNA design for RNA interference Nat. Biotechnol. 22 3 2004 326 330
28. M. Amarzguioui, and H. Prydz An algorithm for selection of functional siRNA sequences Biochem. Biophys. Res. Commun. 316 4 2004 1050 1058
29. A. Peyman, M. Helsberg, G. Kretzschmar, M. Mag, S. Grabley, and E. Uhlmann Inhibition of viral growth by antisense oligonucleotides directed against the IE110 and the UL30 mRNA of herpes simplex virus type-1 Biol. Chem. Hoppe Seyler 376 3 1995 195 198
30. L. Scherer, and J. Rossi Approaches for the sequence-specific knockdown of mRNA Nat. Biotechnol. 21 12 2003 1457 1465
31. P.J. Paddison, A.A. Caudy, E. Bernstein, G.J. Hannon, and D.S. Conklin Short hairpin RNAs (shRNAs) induce sequence-specific silencing in mammalian cells Genes Dev. 16 8 2002 948 958
32. T.R. Brummelkamp, R. Bernards, and R. Agami A system for stable expression of short interfering RNAs in mammalian cells Science 296 5567 2002 550 553
33. Vitravene-Study-Group (2002) A randomized controlled clinical trial of intravitreous fomivirsen for treatment of newly diagnosed peripheral cytomegalovirus retinitis in patients with AIDS. Am. J. Ophthalmol. 133(4), 467-474.
34. E. Check A crucial test Nat. Med. 11 3 2005 243 244
35. Q. Ge, L. Filip, A. Bai, T. Nguyen, H. Eisen, and J. Chen Inhibition of influenza virus production in virus-infected mice by RNA interference Proc. Natl. Acad. Sci. USA 101 23 2004 88676 88681
36. V. Bitko, A. Musiyenko, O. Shulyayeva, and S. Barik Inhibition of respiratory viruses by nasally administered siRNA Nat. Med. 11 1 2005 50 55
37. X. Zhang, P. Shan, D. Jiang, P. Noble, N. Abraham, A. Kappas, and P. Lee Small interfering RNA targeting heme oxygenase-1 enhances ischemia-reperfusion- induced lung apoptosis J. Biol. Chem. 279 11 2004 10677 10684
38. H. Makimura, T. Mizuno, J. Mastaitis, R. Agami, and C. Mobbs Reducing hypothalamic AGRP by RNA interference increases metabolic rate and decreases body weight without influencing food intake BMC Neurosci. 3 1 2002 18
39. G. Dorn, S. Patel, G. Wotherspoon, M. Hemmings-Mieszczak, J. Barclay, F. Natt, P. Martin, S. Bevan, A. Fox, P. Ganju, W. Wishart, and J. Hall siRNA relieves chronic neuropathic pain Nucleic Acids Res. 32 5 2004 e4
40. H. Xia, Q. Mao, S. Eliason, S. Harper, I. Martins, H. Orr, H. Paulson, L. Yang, R. Kotin, and B. Davidson RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia Nat. Med. 10 8 2004 816 820
41. J. Soutschek, A. Akinc, B. Bramlage, K. Charisse, R. Constien, M. Donoghue, S. Elbashir, A. Geick, P. Hadwiger, J. Harborth, M. John, V. Kesavan, G. Lavine, R. Pandey, T. Racie, K. Rajeev, I. Rohl, I. Toudjarska, G. Wang, S. Wuschko, D. Bumcrot, V. Koteliansky, S. Limmer, M. Manoharan, and H. Vornlocher Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs Nature 432 7014 2004 173 178
42. D. Morrissey, K. Blanchard, L. Shaw, K. Jensen, J. Lockridge, B. Dickinson, J. McSwiggen, C. Vargeese, K. Bowman, C. Shaffer, B. Polisky, and S. Zinnen Activity of stabilized short interfering RNA in a mouse model of hepatitis B virus replication Hepatology 41 6 2005 1349 1356
43. M. Sioud, and D. Sorensen Cationic liposome-mediated delivery of siRNAs in adult mice Biochem. Biophys. Res. Commun. 312 4 2003 1220 1225
44. D. Sorensen, M. Leirdal, and M. Sioud Gene silencing by systemic delivery of synthetic siRNAs in adult mice J. Mol. Biol. 327 4 2003 761 766
45. C. Lorenz, P. Hadwiger, M. John, H. Vornlocher, and C. Unverzagt Steroid and lipid conjugates of siRNAs to enhance cellular uptake and gene silencing in liver cells Bioorg. Med. Chem. Lett. 14 19 2004 4975 4977
46. R. Schiffelers, A. Ansari, J. Xu, Q. Zhou, Q. Tang, G. Storm, G. Molema, P. Lu, P. Scaria, and M. Woodle Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle Nucleic Acids Res. 32 19 2004 e149
47. B. Urban-Klein, S. Werth, S. Abuharbeid, F. Czubayko, and A. Aigner RNAi-mediated gene-targeting through systemic application of polyethylenimine (PEI)-complexed siRNA in vivo Gene Ther. 12 5 2005 461 466
48. Y. Zhang, R. Boado, and W. Pardridge In vivo knockdown of gene expression in brain cancer with intravenous RNAi in adult rats J. Gene Med. 5 12 2003 1039 1045
49. Y. Zhang, Y. Zhang, J. Bryant, A. Charles, R. Boado, and W. Pardridge Intravenous RNA interference gene therapy targeting the human epidermal growth factor receptor prolongs survival in intracranial brain cancer Clin. Cancer Res. 10 11 2004 3667 3677
50. W. Pardridge Intravenous, non-viral RNAi gene therapy of brain cancer Expert Opin. Biol. Ther. 4 7 2004 1103 1113
51. E. Song, P. Zhu, S. Lee, D. Chowdhury, S. Kussman, D. Dykxhoorn, Y. Feng, D. Palliser, D. Weiner, P. Shankar, W. Marasco, and J. Lieberman Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors Nat. Biotechnol. 23 6 2005 709 717
52. R. Tomanin, and M. Scarpa Why do we need new gene therapy viral vectors? Characteristics, limitations and future perspectives of viral vector transduction Curr. Gene Ther. 4 4 2004 357 372
53. L. Zentilin, and M. Giacca In vivo transfer and expression of genes coding for short interfering RNAs Curr. Pharm. Biotechnol. 5 4 2004 341 347
54. J. Clayton RNA interference: the silent treatment Nature 431 7008 2004 599 605
55. A. Jackson, S. Bartz, J. Schelter, S. Kobayashi, J. Burchard, M. Mao, B. Li, G. Cavet, and P. Linsley Expression profiling reveals off-target gene regulation by RNAi Nat. Biotechnol. 21 6 2003 635 637
56. Z. Ma, J. Li, F. He, A. Wilson, B. Pitt, and S. Li Cationic lipids enhance siRNA-mediated interferon response in mice Biochem. Biophys. Res. Commun. 330 3 2005 755 759
57. J. Heidel, S. Hu, X. Liu, T. Triche, and M. Davis Lack of interferon response in animals to naked siRNAs Nat. Biotechnol. 22 12 2004 1579 1582
58. D. Semizarov, L. Frost, A. Sarthy, P. Kroeger, D. Halbert, and S. Fesik Specificity of short interfering RNA determined through gene expression signatures Proc. Natl. Acad. Sci. USA 100 11 2003 6347 6352
59. J. Chi, H. Chang, N. Wang, D. Chang, N. Dunphy, and P. Brown Genomewide view of gene silencing by small interfering RNAs Proc. Natl. Acad. Sci. USA 100 11 2003 6343 6346
60. K. Kariko, P. Bhuyan, J. Capodici, and D. Weissman Small interfering RNAs mediate sequence-independent gene suppression and induce immune activation by signaling through toll-like receptor 3 J. Immunol. 172 11 2004 6545 6549
61. C. Sledz, M. Holko, M. de Veer, R. Silverman, and B. Williams Activation of the interferon system by short-interfering RNAs Nat. Cell Biol. 5 9 2003 834 839
62. M. Robbins, and J. Rossi Sensing the danger in RNA Nat. Med. 11 3 2005 250 251
63. V. Hornung, M. Guenthner-Biller, C. Bourquin, A. Ablasser, M. Schlee, S. Uematsu, A. Noronha, M. Manoharan, S. Akira, A. de Fougerolles, S. Endres, and G. Hartmann Sequence-specific potent induction of IFN-alpha by short interfering RNA in plasmacytoid dendritic cells through TLR7 Nat. Med. 11 3 2005 263 270
64. D. Kim, M. Longo, Y. Han, P. Lundberg, E. Cantin, and J. Rossi Interferon induction by siRNAs and ssRNAs synthesized by phage polymerase Nat. Biotechnol. 22 3 2004 321 325
65. G. Hutvagner, and P. Zamore A microRNA in a multiple-turnover RNAi enzyme complex Science 297 5589 2002 2056 2060
66. T. Holen, M. Amarzguioui, M.T. Wiiger, E. Babaie, and H. Prydz Positional effects of short interfering RNAs targeting the human coagulation trigger tissue factor Nucleic Acids Res. 30 8 2002 1757 1766
67. M. McManus, B. Haines, C. Dillon, C. Whitehurst, L. van Parijs, J. Chen, and P. Sharp Small interfering RNA-mediated gene silencing in T lymphocytes J. Immunol. 169 10 2002 5754 5760
68. J. Dostie, Z. Mourelatos, M. Yang, A. Sharma, and G. Dreyfuss Numerous microRNPs in neuronal cells containing novel microRNAs RNA 9 2 2003 180 186
69. R. Carthew RNA interference: the fragile X syndrome connection Curr. Biol. 12 24 2002 R852 R854
70. H. Gong, C. Liu, D. Liu, and C. Liang The role of small RNAs in human diseases: potential troublemaker and therapeutic tools Med. Res. Rev. 25 3 2005 361 381
71. G. Calin, C. Liu, C. Sevignani, M. Ferracin, N. Felli, C. Dumitru, M. Shimizu, A. Cimmino, S. Zupo, M. Dono, M. Dell'Aquila, H. Alder, L. Rassenti, T. Kipps, F. Bullrich, M. Negrini, and C. Croce MicroRNA profiling reveals distinct signatures in B cell chronic lymphocytic leukemias Proc. Natl. Acad. Sci. USA 101 32 2004 11755 11760
72. Y. Karube, H. Tanaka, H. Osada, S. Tomida, Y. Tatematsu, K. Yanagisawa, Y. Yatabe, J. Takamizawa, S. Miyoshi, T. Mitsudomi, and T. Takahashi Reduced expression of Dicer associated with poor prognosis in lung cancer patients Cancer Sci. 96 2 2005 111 115
73. P. Eis, W. Tam, L. Sun, A. Chadburn, Z. Li, M. Gomez, E. Lund, and J. Dahlberg Accumulation of miR-155 and BIC RNA in human B cell lymphomas Proc. Natl. Acad. Sci. USA 102 10 2005 3627 3632
74. S. Johnson, H. Grosshans, J. Shingara, M. Byrom, R. Jarvis, A. Cheng, E. Labourier, K. Reinert, D. Brown, and F. Slack RAS is regulated by the let-7 microRNA family Cell 120 5 2005 635 647
75. J. Lu, G. Getz, E. Miska, E. Alvarez-Saavedra, J. Lamb, D. Peck, A. Sweet-Cordero, B. Ebert, R. Mak, A. Ferrando, J. Downing, T. Jacks, H. Horvitz, and T. Golub MicroRNA expression profiles classify human cancers Nature 435 7043 2005 834 838
76. K. O'Donnell, E. Wentzel, K. Zeller, C. Dang, and J. Mendell c-Myc-regulated microRNAs modulate E2F1 expression Nature 435 7043 2005 839 843
77. L. He, J. Thomson, M. Hemann, E. Hernando-Monge, D. Mu, S. Goodson, S. Powers, C. Cordon-Cardo, S. Lowe, G. Hannon, and S. Hammond A microRNA polycistron as a potential human oncogene Nature 435 7043 2005 828 833
78. D. Boden, O. Pusch, F. Lee, L. Tucker, and B. Ramratnam Human immunodeficiency virus type 1 escape from RNA interference J. Virol. 77 21 2003 11531 11535
79. A. Das, T. Brummelkamp, E. Westerhout, M. Vink, M. Madiredjo, R. Bernards, and B. Berkhout Human immunodeficiency virus type 1 escapes from RNA interference-mediated inhibition J. Virol. 78 5 2004 2601 2605
80. L. Gitlin, J. Stone, and R. Andino Poliovirus escape from RNA interference: short interfering RNA-target recognition and implications for therapeutic approaches J. Virol. 79 2 2005 1027 1035
81. H. Wu, L. Huang, C. Huang, H. Lai, C. Liu, Y. Huang, Y. Hsu, C. Lu, D. Chen, and P. Chen RNA interference-mediated control of hepatitis B virus and emergence of resistant mutant Gastroenterology 128 3 2005 708 716
82. J. Wilson, and C. Richardson Hepatitis C virus replicons escape RNA interference induced by a short interfering RNA directed against the NS5b coding region J. Virol. 79 11 2005 7050 7058
83. P. Pancoska, Z. Moravek, and U. Moll Efficient RNA interference depends on global context of the target sequence: quantitative analysis of silencing efficiency using Eulerian graph representation of siRNA Nucleic Acids Res. 32 4 2004 1469 1479
84. S. Schubert, A. Grunweller, V. Erdmann, and J. Kurreck Local RNA target structure influences siRNA efficacy: systematic analysis of intentionally designed binding regions J. Mol. Biol. 348 4 2005 883 893
85. W. Hu, C. Myers, J. Kilzer, S. Pfaff, and F. Bushman Inhibition of retroviral pathogenesis by RNA interference Curr. Biol. 12 15 2002 1301
86. J. Chang, and J. Taylor Susceptibility of human hepatitis delta virus RNAs to small interfering RNA action J. Virol. 77 17 2003 9728 9731
87. V. Bitko, and S. Barik Phenotypic silencing of cytoplasmic genes using sequence-specific double-stranded short interfering RNA and its application in the reverse genetics of wild type negative-strand RNA viruses BMC Microbiol. 1 1 2001 34
88. U. Verma, R. Surabhi, A. Schmaltieg, C. Becerra, and R. Gaynor Small interfering RNAs directed against beta-catenin inhibit the in vitro and in vivo growth of colon cancer cells Clin. Cancer Res. 9 4 2003 1291 1300
89. G. Yang, J. Thompson, B. Fang, and J. Liu Silencing of H-ras gene expression by retrovirus-mediated siRNA decreases transformation efficiency and tumorgrowth in a model of human ovarian cancer Oncogene 22 36 2003 5694 5701
90. A. Hall, J. Wan, E. Shaughnessy, B. Ramsay Shaw, and K. Alexander RNA interference using boranophosphate siRNAs: structure-activity relationships Nucleic Acids Res. 32 20 2004 5991 6000
91. Y. Chiu, and T. Rana siRNA function in RNAi: a chemical modification analysis RNA 9 9 2003 1034 1048
92. D. Braasch, S. Jensen, Y. Liu, K. Kaur, K. Arar, M. White, and D. Corey RNA interference in mammalian cells by chemically-modified RNA Biochemistry 42 26 2003 7967 7975
93. F. Czauderna, M. Fechtner, S. Dames, H. Aygun, A. Klippel, G. Pronk, K. Giese, and J. Kaufmann Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells Nucleic Acids Res. 31 11 2003 2705 2716
94. P. Joost Haasnoot, D. Cupac, and B. Berkhout Inhibition of virus replication by RNA interference J. Biomed. Sci. 10 6 Pt 1 2003 607 616
95. M. McCown, M. Diamond, and A. Pekosz The utility of siRNA transcripts produced by RNA polymerase i in down regulating viral gene expression and replication of negative- and positive-strand RNA viruses Virology 313 2 2003 514 524
96. F. Bai, T. Wang, U. Pal, F. Bao, L. Gould, and E. Fikrig Use of RNA interference to prevent lethal murine west nile virus infection J. Infect. Dis. 191 7 2005 1148 1154
97. G. Randall, and C. Rice Interfering with hepatitis C virus RNA replication Virus Res. 102 1 2004 19 25
98. A.P. McCaffrey, L. Meuse, T.T. Pham, D.S. Conklin, G.J. Hannon, and M.A. Kay RNA interference in adult mice Nature 418 6893 2002 38 39
99. S. Merl, C. Michaelis, B. Jaschke, M. Vorpahl, S. Seidl, and R. Wessely Targeting 2A protease by RNA interference attenuates coxsackieviral cytopathogenicity and promotes survival in highly susceptible mice Circulation 111 13 2005 1583 1592
100. Q. Ge, M. McManus, T. Nguyen, C. Shen, P. Sharp, H. Eisen, and J. Chen RNA interference of influenza virus production by directly targeting mRNA for degradation and indirectly inhibiting all viral RNA transcription Proc. Natl. Acad. Sci. USA 100 5 2003 2718 2723
101. Q. Ge, H. Eisen, and J. Chen Use of siRNAs to prevent and treat influenza virus infection Virus Res. 102 1 2004 37 42
102. S. Tompkins, C. Lo, T. Tumpey, and S. Epstein Protection against lethal influenza virus challenge by RNA interference in vivo Proc. Natl. Acad. Sci. USA 101 23 2004 8682 8686
103. C. Ying, E. De Clercq, and J. Neyts Selective inhibition of hepatitis B virus replication by RNA interference Biochem. Biophys. Res. Commun. 309 2 2003 482 484
104. K. Hamasaki, K. Nakao, K. Matsumoto, T. Ichikawa, H. Ishikawa, and K. Eguchi Short interfering RNA-directed inhibition of hepatitis B virus replication FEBS Lett. 543 1-3 2003 51 54
105. A. Shlomai, and Y. Shaul Inhibition of hepatitis B virus expression and replication by RNA interference Hepatology 37 4 2003 764 770
106. A. McCaffrey, H. Nakai, K. Pandey, Z. Huang, F. Salazar, H. Xu, S. Wieland, P. Marion, and M. Kay Inhibition of hepatitis B virus in mice by RNA interference Nat. Biotechnol. 21 6 2003 639 644
107. H. Giladi, M. Ketzinel-Gilad, L. Rivkin, Y. Felig, O. Nussbaum, and E. Galun Small interfering RNA inhibits hepatitis B virus replication in mice Mol. Ther. 8 5 2003 767 769
108. Y. Zhang, T. Li, L. Fu, C. Yu, Y. Li, X. Xu, Y. Wang, H. Ning, S. Zhang, W. Chen, L. Babiuk, and Z. Chang Silencing SARS-CoV Spike protein expression in cultured cells by RNA interference FEBS Lett. 560 1-3 2004 141 146
109. K. Phipps, A. Martinez, J. Lu, B. Heinz, and G. Zhao Small interfering RNA molecules as potential anti-human rhinovirus agents: in vitro potency, specificity, and mechanism Antiviral Res. 61 1 2004 49 55
110. L. Gitlin, S. Karelsky, and R. Andino Short interfering RNA confers intracellular antiviral immunity in human cells Nature 418 6896 2002 430 434
111. B. Berkhout RNA interference as an antiviral approach: targeting HIV-1 Curr. Opin. Mol. Ther. 6 2 2004 141 145
112. M. Jiang, and J. Milner Selective silencing of viral gene expression in HPV-positive human cervical carcinoma cells treated with siRNA, a primer of RNA interference Oncogene 21 39 2002 6041 6048
113. L. Wiebusch, M. Truss, and C. Hagemeier Inhibition of human cytomegalovirus replication by small interfering RNAs J. Gen. Virol. 85 Pt 1 2004 179 184
114. S. Radhakrishnan, T. Layden, and A. Gartel RNA interference as a new strategy against viral hepatitis Virology 323 2 2004 173 181
115. P. Arbuthnot, S. Carmona, and A. Ely Exploiting the RNA interference pathway to counter hepatitis B virus replication Liver Int. 25 1 2005 9 15
116. L.G. Guidotti, B. Matzke, H. Schaller, and F.V. Chisari High level hepatitis B virus replication in transgenic mice J. Virol. 69 1995 6158 6169
117. P.L. Yang, A. Althage, J. Chung, and F.V. Chisari Hydrodynamic injection of viral DNA: a mouse model of acute hepatitis B virus infection Proc. Natl. Acad. Sci. USA 99 21 2002 13825 13830
118. C. Klein, C. Bock, H. Wedemeyer, T. Wustefeld, S. Locarnini, H. Dienes, S. Kubicka, M. Manns, and C. Trautwein Inhibition of hepatitis B virus replication in vivo by nucleoside analogues and siRNA Gastroenterology 125 1 2003 9 18
119. C.D. Novina, M.F. Murray, D.M. Dykxhoorn, P.J. Beresford, J. Riess, S.K. Lee, R.G. Collman, J. Lieberman, P. Shankar, and P.A. Sharp siRNA-directed inhibition of HIV-1 infection Nat. Med. 8 7 2002 681 686
120. X. Qin, D. An, I. Chen, and D. Baltimore Inhibiting HIV-1 infection in human T cells by lentiviral-mediated delivery of small interfering RNA against CCR5 Proc. Natl. Acad. Sci. USA 100 1 2003 183 188
121. E. Song, S. Lee, D. Dykxhoorn, C. Novina, D. Zhang, K. Crawford, J. Cerny, P. Sharp, J. Lieberman, N. Manjunath, and P. Shankar Sustained small interfering RNA-mediated human immunodeficiency virus type 1 inhibition in primary macrophages J. Virol. 77 13 2003 7174 7181
122. H. Arteaga, J. Hinkula, I. van Dijk-Hard, M. Dilber, B. Wahren, B. Christensson, A. Mohamed, and C. Smith Choosing CCR5 or Rev siRNA in HIV-1 Nat. Biotechnol. 21 3 2003 230 231
123. M. Martinez, A. Gutierrez, M. Armand-Ugon, J. Blanco, M. Parera, J. Gomez, B. Clotet, and J. Este Suppression of chemokine receptor expression by RNA interference allows for inhibition of HIV-1 replication AIDS 16 18 2002 2385 2390
124. S. Buckingham, B. Esmaeili, M. Wood, and D. Sattelle RNA interference: from model organisms towards therapy for neural and neuromuscular disorders Hum. Mol. Genet. 13 2 2004 R275 R288
125. M. Wood, B. Trulzsch, A. Abdelgany, and D. Beeson Therapeutic gene silencing in the nervous system Hum. Mol. Genet. 12 2 2003 R279 R284
126. G. Ralph, P. Radcliffe, D. Day, J. Carthy, M. Leroux, D. Lee, L. Wong, L. Bilsland, L. Greensmith, S. Kingsman, K. Mitrophanous, N. Mazarakis, and M. Azzouz Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model Nat. Med. 11 4 2005 429 433
127. C. Raoul, T. Abbas-Terki, J. Bensadoun, S. Guillot, G. Haase, J. Szulc, C. Henderson, and P. Aebischer Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS Nat. Med. 11 4 2005 423 428
128. S. Harper, P. Staber, X. He, S. Eliason, I. Martins, Q. Mao, L. Yang, R. Kotin, H. Paulson, and B. Davidson RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model Proc. Natl. Acad. Sci. USA 102 16 2005 5820 5825
129. H. Nakamura, S. Siddiqui, X. Shen, A. Malik, J. Pulido, N. Kumar, and B. Yue RNA interference targeting transforming growth factor-beta type II receptor suppresses ocular inflammation and fibrosis Mol. Vis. 10 10 2004 703 711
130. S. Reich, J. Fosnot, A. Kuroki, W. Tang, X. Yang, A. Maguire, J. Bennett, and M. Tolentino Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in a mouse model Mol. Vis. 9 2003 210 216
131. B. Kim, Q. Tang, P. Biswas, J. Xu, R. Schiffelers, F. Xie, A. Ansari, P. Scaria, M. Woodle, P. Lu, and B. Rouse Inhibition of ocular angiogenesis by siRNA targeting vascular endothelial growth factor pathway genes: therapeutic strategy for herpetic stromal keratitis Am. J. Pathol. 156 6 2004 2177 2185
132. Y. Maeda, K. Fukushima, K. Nishizaki, and R. Smith In vitro and in vivo suppression of GJB2 expression by RNA interference Hum. Mol. Genet. 14 12 2005 1641 1650
133. R. Schiffelers, J. Xu, G. Storm, M. Woodle, and P. Scaria Effects of treatment with small interfering RNA on joint inflammation in mice with collagen-induced arthritis Arthritis Rheum. 52 4 2005 1314 1318
134. E. Song, S. Lee, J. Wang, N. Ince, N. Ouyang, J. Min, J. Chen, P. Shankar, and J. Lieberman RNA interference targeting Fas protects mice from fulminant hepatitis Nat. Med. 9 3 2003 347 351
135. L. Zender, S. Hutker, C. Liedtke, H. Tillmann, S. Zender, B. Mundt, M. Waltemathe, T. Gosling, P. Flemming, N. Malek, C. Trautwein, M. Manns, F. Kuhnel, and S. Kubicka Caspase 8 small interfering RNA prevents acute liver failure in mice Proc. Natl. Acad. Sci. USA 100 13 2003 7797 7802
136. J. Contreras, M. Vilatoba, C. Eckstein, G. Bilbao, J. Anthony Thompson, and D. Eckhoff Caspase-8 and caspase-3 small interfering RNA decreases ischemia/reperfusion injury to the liver in mice Surgery 136 2 2004 390 400
137. P. Hamar, E. Song, G. Kokeny, A. Chen, N. Ouyang, and J. Lieberman Small interfering RNA targeting Fas protects mice against renal ischemia-reperfusion injury Proc. Natl. Acad. Sci. USA 101 41 2004 14883 14888
138. L. Rowland, and N. Shneider Amyotrophic lateral sclerosis N. Engl. J. Med. 344 22 2001 1688 1700
139. X. Xia, H. Zhou, S. Zhou, Y. Yu, R. Wu, and Z. Xu An RNAi strategy for treatment of amyotrophic lateral sclerosis caused by mutant Cu,Zn superoxide dismutase J. Neurochem. 92 2 2005 362 367
140. E. Burright, H. Clark, A. Servadio, T. Matilla, R. Feddersen, W. Yunis, L. Duvick, H. Zoghbi, and H. Orr SCA1 transgenic mice: a model for neurodegeneration caused by an expanded CAG trinucleotide repeat Cell 82 6 1995 937 948
141. L. Mangiarini, K. Sathasivam, M. Seller, B. Cozens, A. Harper, C. Hetherington, M. Lawton, Y. Trottier, H. Lehrach, S. Davies, and G. Bates Exon 1 of the HD gene with an expanded CAG repeat is sufficient to cause a progressive neurological phenotype in transgenic mice Cell 87 3 1996 493 506
142. T. Zu, L. Duvick, M. Kaytor, M. Berlinger, H. Zoghbi, H. Clark, and H. Orr Recovery from polyglutamine-induced neurodegeneration in conditional SCA1 transgenic mice J. Neurosci. 24 40 2004 8853 8861
143. S. Kao, A. Krichevsky, K. Kosik, and L. Tsai BACE1 suppression by RNA interference in primary cortical neurons J. Biol. Chem. 279 3 2004 1942 1949
144. U. Fuchs, C. Damm-Welk, and A. Borkhardt Silencing of disease-related genes by small interfering RNAs Curr. Mol. Med. 4 5 2004 507 517
145. J. Yano, K. Hirabayashi, S. Nakagawa, T. Yamaguchi, M. Nogawa, I. Kashimori, H. Naito, H. Kitagawa, K. Ishiyama, T. Ohgi, and T. Irimura Antitumor activity of small interfering RNA/cationic liposome complex in mouse models of cancer Clin. Cancer Res. 10 22 2004 7721 7726
146. Z. Liang, Y. Yoon, J. Votaw, M. Goodman, L. Williams, and H. Shim Silencing of CXCR4 blocks breast cancer metastasis Cancer Res. 65 3 2005 967 971
147. M. Duxbury, H. Ito, E. Benoit, M. Zinner, S. Ashley, and E. Whang RNA interference targeting focal adhesion kinase enhances pancreatic adenocarcinoma gemcitabine chemosensitivity Biochem. Biophys. Res. Commun. 311 3 2003 786 792
148. M. Duxbury, H. Ito, M. Zinner, S. Ashley, and E. Whang EphA2: a determinant of malignant cellular behavior and a potential therapeutic target in pancreatic adenocarcinoma Oncogene 23 7 2004 1448 1456
149. B. Spankuch, Y. Matthess, R. Knecht, B. Zimmer, M. Kaufmann, and K. Strebhardt Cancer inhibition in nude mice after systemic application of U6 promoter-driven short hairpin RNAs against PLK1 J. Natl. Cancer Inst. 96 11 2004 862 872
150. S. Aharinejad, P. Paulus, M. Sioud, M. Hofmann, K. Zins, R. Schafer, E. Stanley, and D. Abraham Colony-stimulating factor-1 blockade by antisense oligonucleotides and small interfering RNAs suppresses growth of human mammary tumor xenografts in mice Cancer Res. 64 15 2004 5378 5384
151. H. Caldas, M. Holloway, B. Hall, S. Qualman, and R. Altura Survivin-directed RNA interference cocktail is a potent suppressor of tumor growth in vivo J. Med. Genet. 2005
152. M. Duxbury, E. Matros, H. Ito, M. Zinner, S. Ashley, and E. Whang Systemic siRNA-mediated gene silencing: a new approach to targeted therapy of cancer Ann. Surg. 240 4 2004 667 674
153. H. Sumimoto, S. Yamagata, A. Shimizu, H. Miyoshi, H. Mizuguchi, T. Hayakawa, M. Miyagishi, K. Taira, and Y. Kawakami Gene therapy for human small-cell lung carcinoma by inactivation of Skp-2 with virally mediated RNA interference Gene Ther. 12 1 2005 95 100
154. S. Chae, J. Paik, H. Furneaux, and T. Hla Requirement for sphingosine 1-phosphate receptor-1 in tumor angiogenesis demonstrated by in vivo RNA interference J. Clin. Invest. 114 8 2004 1082 1089
155. J. Pille, C. Denoyelle, J. Varet, J. Bertrand, J. Soria, P. Opolon, H. Lu, L. Pritchard, J. Vannier, C. Malvy, C. Soria, and H. Li Anti-RhoA and anti-RhoC siRNAs inhibit the proliferation and invasiveness of MDA-MB-231 breast cancer cells in vitro and in vivo Mol. Ther. 11 2 2005 267 274
156. Y. Takei, K. Kadomatsu, Y. Yuzawa, S. Matsuo, and T. Muramatsu A small interfering RNA targeting vascular endothelial growth factor as cancer therapeutics Cancer Res. 64 10 2004 3365 3370
157. Y. Minakuchi, F. Takeshita, N. Kosaka, H. Sasaki, Y. Yamamoto, M. Kouno, K. Honma, S. Nagahara, K. Hanai, A. Sano, T. Kato, M. Terada, and T. Ochiya Atelocollagen-mediated synthetic small interfering RNA delivery for effective gene silencing in vitro and in vivo Nucleic Acids Res. 32 13 2004 e109
158. D. Cioca, Y. Aoki, and K. Kiyosawa RNA interference is a functional pathway with therapeutic potential in human myeloid leukemia cell lines Cancer Gene Ther. 10 2 2003 125 133
159. L. Martinez, I. Naguibneva, H. Lehrmann, A. Vervisch, T. Tchenio, G. Lozano, and A. Harel-Bellan Synthetic small inhibiting RNAs: efficient tools to inactivate oncogenic mutations and restore p53 pathways Proc. Natl. Acad. Sci. USA 99 23 2002 14849 14854
160. U. Zangemeister-Wittke Antisense to apoptosis inhibitors facilitates chemotherapy and TRAIL-induced death signaling Ann. NY Acad. Sci. 1002 2003 90 94
161. T. Futami, M. Miyagishi, M. Seki, and K. Taira Induction of apoptosis in HeLa cells with siRNA expression vector targeted against bcl-2 Nucleic Acids Res. Suppl. 2002 2 2002 251 252
162. S. Collis, M. Swartz, W. Nelson, and T. DeWeese Enhanced radiation and chemotherapy-mediated cell killing of human cancer cells by small inhibitory RNA silencing of DNA repair factors Cancer Res. 63 7 2003 1550 1554
163. Y. Peng, Q. Zhang, H. Nagasawa, R. Okayasu, H. Liber, and J. Bedford Silencing expression of the catalytic subunit of DNA-dependent protein kinase by small interfering RNA sensitizes human cells for radiation-induced chromosome damage, cell killing, and mutation Cancer Res. 62 22 2002 6400 6404
164. T. Tsuruo, M. Naito, A. Tomida, N. Fujita, T. Mashima, H. Sakamoto, and N. Haga Molecular targeting therapy of cancer: drug resistance, apoptosis and survival signal Cancer Sci. 94 1 2003 15 21
165. C. Nieth, A. Priebsch, A. Stege, and H. Lage Modulation of the classical multirug resistance (MDR) phenotype by RNA interference (RNAi) FEBS Lett. 545 2-3 2003 144 150
166. E. Yague, C. Higgins, and S. Raguz Complete reversal of multidrug resistance by stable expression of small interfering RNAs targeting MDR1 Gene Ther. 11 14 2004 1170 1174
167. N. Lapteva, A. Yang, D. Sanders, R. Strube, and S. Chen CXCR4 knockdown by small interfering RNA abrogates breast tumor growth in vivo Cancer Gene Ther. 12 1 2005 84 89
168. Y. Chen, G. Stamatoyannopoulos, and C. Song Down-regulation of CXCR4 by inducible small interfering RNA inhibits breast cancer cell invasion in vitro Cancer Res. 63 16 2003 4801 4804
169. P. Lu, F. Xie, and M. Woodle Modulation of angiogenesis with siRNA inhibitors for novel therapeutics Trends Mol. Med. 11 3 2005 104 113
170. S. Filleur, A. Courtin, S. Ait-Si-Ali, J. Guglielmi, C. Merle, A. Harel-Bellan, P. Clezardin, and F. Cabon SiRNA-mediated inhibition of vascular endothelial growth factor severely limits tumor resistance to antiangiogenic thrombospondin-1 and slows tumor vascularization and growth Cancer Res. 63 14 2003 3919 3922
171. E. Ng, and A. Adamis Targeting angiogenesis, the underlying disorder in neovascular age-related macular degeneration Can. J. Ophthalmol. 40 3 2005 352 368
172. S. Fine, D. Martin, and P. Kirkpatrick Pegaptanib sodium Nat. Rev. Drug Discov. 4 3 2005 187 188
173. M. Hochberg, M. Lebwohl, S. Plevy, K. Hobbs, and D. Yocum The benefit/risk profile of TNF-blocking agents: findings of a consensus panel Semin. Arthritis Rheum. 34 6 2005 819 836