Short interfering RNAs as a tool for cancer gene therapy

Cancer Gene Therapy

Volumn 12, Issue 3, 2005, Pages 217-227

  Izquierdo, Marta ^a  

^a UNIVERSIDAD AUTÓNOMA DE MADRID   (Spain)

Author keywords

Gene target; Review; RNAi; siRNA

Indexed keywords

CYCLIN E; DOUBLE STRANDED RNA; ENDONUCLEASE; MICRORNA; RIBONUCLEASE III; SMALL INTERFERING RNA;

ANIMAL MODEL; APOPTOSIS; CANCER THERAPY; CELL DIVISION; GENE EXPRESSION; GENE SILENCING; GENE TARGETING; GENE THERAPY; MULTIDRUG RESISTANCE; NONHUMAN; ONCOGENE; PRIORITY JOURNAL; RAT; REVIEW; RNA CLEAVAGE;

APOPTOSIS; CELL CYCLE; GENE EXPRESSION REGULATION, NEOPLASTIC; GENE THERAPY; GENES, MDR; GENETIC VECTORS; NEOPLASMS; RNA INTERFERENCE; RNA, MESSENGER; RNA, SMALL INTERFERING;

ANIMALIA;

EID: 14644435784     PISSN: 09291903     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.cgt.7700791     Document Type: Review

References (118)

1. 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;2:279-289.
2. Van der Krol AR, Mur LA, Beld M, et al. Flavonoid genes in Petunia: addition of a limited number of gene copies may lead to a suppression of gene expression. Plant Cell. 1990;2:291-299.
3. Fire A, Xu S, Montgomery MK, et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature. 1998;391:806-811.
4. Tuschl T, Zamore PD, Lehman R, et al. Targeted mRNA degradation by double-stranded RNA in vitro. Genes Dev. 1999;15:3191-3197.
5. Hammond S, Bernstein E, Beach D, et al. An RNA-directed nuclease mediates post-transcriptional gene silencing in Drosophila cells. Nature. 2000;404:293-296.
6. Nicanen A, Haley B, Zamore PD. ATP requirements and small interfering RNA structure in the RNA interference pathway. Cell. 2001;107:309-321.
7. Schwarz DS, Hutvagner G, Haley B, et al. Evidence that siRNAs function as guides, not primers, in the Drosophila and human RNAi pathways. Mol Cell. 2002;10:537-548.
8. Elbashir SM, Lendeckel W, Tuschi T. RNA interference is mediated by 21- and 22-nucleotide RNAs. Genes Dev. 2001;15:188-200.
9. Dalmay T, Hamilton A, Rudd S, et al. An RNA dependent RNA polymerase in Arabidopsis is required for posttranscriptional gene silencing mediated by a transgene but not by a virus. Cell. 2000;101:543-553.
10. Cogoni C, Macino G. Gene silencing in Neurospora crassa requires a protein homologous to RNA-dependent RNA polymerase. Nature. 1999;399:166-169.
11. Smardon A, Spoerke JM, Stacey SC, et al. EGO-1 is related to RNA-directed RNA polymerase and functions in germline development and RNA interference in C. elegans. Curr Biol. 2000;10:169-178.
12. Li H, Li WX, Ding SW. Induction and suppression of RNA silencing by an animal virus. Science. 2002;296:1319-1321.
13. Lagos-Quintana M, Rauhut R, Lendeckel W, et al. Identification of novel genes coding for small expressed RNAs. Science. 2001;294:853-858.
14. Lau NC, Lim LP, Weinstein EG, et al. An abundant class of tiny RNAs with probable regulatory roles in Caenorhabditis elegans. Science. 2001;294:858-862.
15. Bartel DP. MicroRNAs: genomics, biogenesis mechanism, and function. Cell. 2004;116:281-297.
16. Bartel B, Bartel DP. MicroRNAs: at the root of plant development? Plant Physiol. 2003;132:709-717.
17. Chen CZ, Li L, Lodish HF, et al. MicroRNAs modulate hematopoietic lineage differentiation. Science. 2004;303:83-86.
18. Calin GA, Sevignani C, Dumitru CD, et al. Human microRNA genes are frequently located at fragile sites and genomic regions involved in cancers. Proc Natl Acad Sci USA. 2004;101:2999-3004.
19. Basyuk E, Suavet F, Doglio A, et al. Human let-7 stem-loop precursors harbor features of RNase III cleavage products. Nucleic Acid Res. 2003;31:6593-6597.
20. Lund E, Güttinger S, Calado A, et al. Nuclear export of microRNA precursors. Science. 2004;303:95-98.
21. Yi R, Qin Y, Macara IG, et al. Exportin-5 mediates the nuclear export of pre-microRNAs and short hairpin RNAs. Genes Dev. 2003;17:3011-3016.
22. Lee Y, Ahn C, Han J, et al. The nuclear RNase Ill Drosha initiates microRNA processing. Nature. 2003;425:415-419.
23. Zamore PD, Tuschl T, Sharp PA, et al. RNAi:double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals. Cell. 2000;101:25-33.
24. Bernstein E, Caudy AA, Hammond SM, et al. Role of a bidentate ribonuclease in the initiation step of RNA interference. Nature. 2001;409:295-296.
25. Zhang H, Kolb FA, Brondani V, et al. Human Dicer preferentially cleaves dsRNAs at their termini without a requirement for ATP. EMBO J. 2002;21:5875-5885.
26. Chen CZ, Li L, Lodish HF, et al. MicroRNAs modulate hematopoietic lineage differentiation. Science. 2004;303:83-86.
27. Zeng Y, Yi R, Cullen BR. MicroRNAs and small interfering RNAs can inhibit mRNA expression by similar mechanisms. Proc Natl Acad Sci USA. 2003;100:9779-9784.
28. Doench JG, Peterson CP, Sharp PA. siRNAs can function as miRNAs. Genes Dev. 2003;17:438-442.
29. Pal-Bhadra M, Leibovitch BA, Sumit GG, et al. Heterochromatic silencing and HPI localization in Drosophila are dependent on the RNAi machinery. Science. 2004;303:669-672.
30. Verdel A, Jia S, Gerber S, et al. RNAi-mediated targeting of heterochromatin by the RITS complex. Science. 2004;303:672-676.
31. Aukerman MJ, Sakai H. Regulation of flowering time and floral organ identity by a MicroRNA and its APETALA2-like target genes. Plant Cell. 2003;10:10.
32. Marathe R, Anandalakshmi R, Smith TH, et al. RNA viruses as inducers, suppressors and targets of posttranscriptional gene silencing. Plant Mol Biol. 2000;43:295-306.
33. Vance V, Vaucheret H. RNA silencing in plants - defense and counterdefense. Science. 2001;292:2277-2280.
34. Waterhouse PM, Helliwell CA. Exploring plant genomes by RNA-induced gene silencing. Nat Rev Genet. 2003;4:29-38.
35. Lee YS, Nakahara K, Pham JW, et al. Distinct roles for Drosophila Dicer-1 and Dicer-2 in the siRNA/miRNA silencing pathways. Cell. 2004;117:69-81.
36. Finnegan EJ, Margis R, Waterhouse PM. Posttranscriptional gene silencing is not compromised in the Arabidopsis CARPEL FACTORY (DICER-LIKE1) mutant, a homolog of Dicer-1 from Drosophila. Curr Biol. 2003;13:236-240.
37. Pham JW, Pellino JL, Lee YS, et al. A Dicer-2-dependent 80S complex cleaves targeted mRNAs during RNAi in Drosophila. 2004;117:83-94.
38. Sijen T, Plasterk RH. Transposon silencing in the Caenorhabditis elegans germ line by natural RNAi. Nature. 2003;426:310-314.
39. Reinhart BJ, Slack FJ, Basson M, et al. The 21 nucleotide let-7 RNA regulates developmental timing in Caenorhabditis elegans. Nature. 2000;403:901-906.
40. Brennecke J, Hipfner DR, Stark A, et al. Bantam encodes a developmentally regulated microRNA that controls cell proliferation and regulates the proapoptotic gene hid in Drosophila. Cell. 2003;113:25-36.
41. Calin GA, Sevignani C, Dumitru CD, et al. Human microRNA genes are frequently located at fragile sites and genomic regions involved in cancers. Proc Natl Acad Sci USA. 2004;101:2999-3004.
42. Ambros V, Lee RC. Identification of microRNAs and other tiny noncoding RNAs by cDNA cloning. Methods Mol Biol. 2004;265:131-158.
43. Kuwabara T, Hsieh J, Nakashima K, et al. A small modulatory dsRNA specifies the fate of adult neural stem cells. Cell. 2004;116:779-793.
44. Novina CD, Sharp PA. The RNAi revolution. Nature. 2004;430:161-164.
45. Bridge AJ, Pebernard S, Ducraux A, et al. Induction of an interferon response by RNAi vectors in mammalian cells. Nat Genet. 2003;34:363-364.
46. Sledz CA, Holko M, de Veer MJ, et al. Activation of the interferon system by short-interfering RNAs. Nat Cell Biol. 2003;5:834-839.
47. Stojdl DF, Lichty B, Knowles S, et al. Exploiting tumor-specific defects in the interferon pathway with a previously unknown oncolytic virus. Nat Med. 2000;6:821-825.
48. Yoshinari K, Miyagishi M, Taira K. Effects on RNAi of the tight structure, sequence and position of the targeted region. Nucleic Acid Res. 2004;32:691-699.
49. Khvorova A, Reynolds A, Jayasena SD. Functional siRNAs and miRNAs exhibit strand bias. Cell. 2003;115:209-216.
50. Schwarz DS, Hutvagner G, Du T, et al. Asymmetry in the assembly of the RNAi enzyme complex. Cell. 2003;115:199-208.
51. Reynolds A, Leake D, Boese Q, et al. Rational siRNA design for RNA interference. Nat Biotechnol. 2004;22:326-330.
52. Ui-Tei K, Naito Y, Takahashi F, et al. Guidelines for the selection of highly effective siRNA sequences for mammalian and chick RNA interference. Nucleic Acids Res. 2004;32:936-948.
53. Meister G, Landthaler M, Dorsett Y, et al. Sequence-specific inhibition of microRNA- and siRNA-induced RNA silencing. RNA. 2004;10:544-550.
54. Dunoyer P, Lecellier CH, Parizotto EA, et al. Probing the microRNA and small interfering RNA pathways with virus-encoded suppressors of RNA silencing. Plant Cell. 2004;5:1235-1250.
55. Zamore PD. Plant RNAi: how a viral silencing suppressor inactivates siRNA. Curr Biol. 2004;14:R198-R200.
56. Elbashir SM, Harborth J, Lendeckel W, et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature. 2001;411:494-498.
57. Kim DH, Longo M, Han Y, et al. Interferon induction by siRNAs and ssRNAs synthesized by phage polymerase. Nat Biotechnol, 2004;22:321-325.
58. Semizarov D, Frost L, Sarthy A, et al. Specificity of short interfering RNA determined through gene expression signatures. Proc Natl Acad Sci USA. 2003;100:6347-6352.
59. Scacheri PC, Rozenblatt-Rosen O, Caplen NJ, 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;101:1892-1897.
60. Brummelkamp TR, Bernards R, Agami R. A system for stable expression of short interfering RNAs in mammalian cells. Science. 2002;296:550-553.
61. Sui G, Soohoo C, Affar E, et al. A DNA vector-based RNAi technology to suppress gene expression in mammalian cells. Proc Natl Acad Sci USA. 2002;99:5515-5520.
62. Shinagawa T, Ishii S. Generation of Ski-knockdown mice by expressing a long double-strand RNA from an RNA polymerase II promoter. Genes Dev. 2003;17:1340-1345.
63. Brummelkamp TR, Bernards R, Agami R. Stable suppression of tumorigenicity by virus-mediated RNA interference. Cancer Cell. 2002;2:243-247.
64. Devroe E, Silver PA. Retrovirus-delivered siRNA. BMC Biotechnol. 2002;2:15-20.
65. de Felipe P, Izquierdo M. Tricistronic and tetracistronic retroviral vectors for gene transfer. Hum Gene Ther. 2000;11:1921-1931.
66. Hawkins LK, Lemoine NR, Kirn D. Oncolytic biotherapy: a novel therapeutic platform. Lancet Oncol. 2002;3:17-26.
67. Freytag SO, Khil M, Stricker H, et al. Phase 1 study of replication-competent adenovirus-mediated double suicide gene therapy for the treatment of locally recurrent prostate cancer. Cancer Res. 2002;62:4968-4976.
68. Carette JE, Overmeer RM, Schagen FHE, et al. Conditionally replicating adenoviruses expressing short hairpin RNAs silence the expression of a target gene in cancer cells. Cancer Res. 2004;64:2663-2667.
69. Rubinson DA, Dillon CP, Kwiatkowski AV, et al. A lentivirus-based system to functionally silence genes in primary mammalian cells, stem cells and transgenic mice by RNA interference. Nat. Genet. 2003;33:401-406.
70. Hommel JD, Sears RM, Georgescu D, et al. Local gene knockdown in the brain using viral-mediated RNA interference. Nat Med. 3003;9:1539-1544.
71. Van Den Haute C, Eggermont K, Nuttin B, et al. Lentiviral vector-mediated delivery of short hairpin RNA results in persistent knockdown of gene expression in mouse brain. Hum Gene Ther. 2003;14:1799-1807.
72. V599E in human melanoma abrogates transformation. Cancer Res. 2003;63:5198-5202.
73. Sumimoto H, Miyagishi M, Miyoshi H, et al. Inhibition of growth and invasive ability of melanoma by inactivation of mutated BRAF with lentivirus-mediated RNA interference. Oncogene. 2004;24 advanced publication 21 June.
74. Mitsushita J, Lambeth JD, Kamata T. The superoxide-generating oxidase Nox1 is functionally required for Ras oncogene transformation. Cancer Res. 2004;64:3580-3585.
75. Davies H, Bignell GR, Cox C, et al. Mutations of the BRAF gene in human cancer. Nature. 2002;417:949-954.
76. Lyons JF, Wilhelm S, Hibner B, et al. Discovery of a novel Raf kinase inhibitor. Endocr-Relat Cancer. 2001;8:219-225.
77. Menendez JA, Vellon L, Mehmi I, et al. Inhibition of fatty acid synthase (FAS) suppresses HER2/neu (erbB-2) oncogene overexpression in cancer cells. Proc Natl Acad Sci USA. 2004, www.pnas.org/cgi/doi/10.1073/pnas.0403390101 online early edition.
78. Li K, Lin S, Brunicardi FC, et al. Use of RNA interference to target cyclin E-overexpressing hepatocellular carcinoma. Cancer Res. 2003;63:3593-3597.
79. Martin-Lluesma S, Stucke VM, Nigg EA. Role of Hec1 in spindle checkpoint signalling and kinetochore recruitment of Mad1/Mad2. Science. 2002;297:2267-2270.
80. Chen Y, Riley DJ, Zheng L, et al. Phosphorylation of the mitotic regulator protein Hec1 by Nek2 kinase is essential for faithful chromosome segregation. J Biol Chem. 2002;277:49408-49416.
81. Cohen M, Feinstein N, Wilson KL, et al. Nuclear pore protein gp210 is essential for viability in Hela cells and Caenorhabditis elegans. Mol Biol Cell. 2003;14:4230-4237.
82. Duensing A, Medeiros F, McConarty B, et al. Mechanisms of oncogenic KIT signal transduction in primary gastrointestinal stromal tumors (GISTs). Oncogene. 2004;23:3999-4006.
83. Demetri GD, von Mehren M, Blanke CD, et al. Efficacy and safety of imatinib mesylate in advance gastrointestinal stromal tumors. N Engl J Med. 2002;347:472-480.
84. Wilda M, Fuchs U, Wossmann W, et al. Killing of leukemic cells with a BCR/ABL fusion gene by RNA interference (RNAi). Oncogene. 2002;21:5716-5724.
85. Scherr M, Battmer K, Winkler T, et al. Specific inhibition of bcr-abl gene expression by small interfering RNA. Blood. 2003;101:1566-1569.
86. Nieth C, Priebsch A, Stege A, et al. Modulation of the classical multidrug resistance (MDR) phenotype by RNA interference (RNAi). FEBS Lett. 2003;545:144-150.
87. Duxbury MS, Ito H, Zinner MJ, et al. RNA interference targeting the M2 subunit of ribonucleotide reductase enhances pancreatic adenocarcinoma chemosensitivity to gemcitabine. Oncogene. 2004;23:1539-1548.
88. Daniel JC, Cao X, Miller SD, et al. RNA interference of the bcl-xl gene in esophageal adenocarcinoma. J Surg Res. 2003;114:278.
89. Crnkovic-Mertens I, Hoppe-Seyler F, Butz K. Induction of apoptosis in tumor cells by siRNA-mediated silencing of the livin/ML-IAP/KIAP gene. Oncogene. 2003;22:8330-8336.
90. Altieri DC. Survivin, versatile modulation of cell division and apoptosis in cancer. Oncogene. 2003;22:8581-8589.
91. Carvalho A, Carmena M, Sambade C. Survivin is required for stable checkpoint activation in taxol-treated Hela cells. J Cell Sci. 2003;116:2987-2998.
92. Ling X, Li F. Silencing of antiapoptotic and surviving gene by multiple approaches of RNA interference technology. Biotechniques. 2004;36:450-460.
93. Brower V. Tumor angiogenesis - new drugs on the block. Nat Biotechnol. 1999;17:963-968.
94. Jansen M, de Witt Hamer PC, Witmer AN, et al. Current perspectives on antiangiogenesis strategies in the treatment of malignant gliomas. Brain Res Rev. 2004;45:143-163.
95. Novak K. Angiogenesis inhibitors revised and revived at AACR. American Association for Cancer Research. Nat Med. 2002;8:427.
96. Filleur S, Volpert O, Degeorges A, et al. In vivo mechanisms by which tumors producing thrombospondin 1 bypass its inhibitory effects. Genes Dev. 2001;15:1373-1382.
97. Filleur S, Courtin A, Ait-Si-Ali S, 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;63:3919-3922.
98. Cullen BR, Lomedico PT, Ju G. Transcriptional interference in avian retroviruses-implications for the promoter insertion model of leukaemogenesis. Nature. 1984;307:241-245.
99. Emmerman M, Temin HM. High-frequency deletion in recovered retrovirus vector containing endogenous DNA with promoters. J Virol. 1984;50:42-49.
100. Emmerman M, Temin HM. Genes with promoters in retrovirus vectors can be independently suppressed by an epigenetic mechanism. Cell. 1984;39:459-467.
101. Emmerman M, Temin HM. Quantitative analysis of gene suppression in retrovirus vectors. Mol Cell Biol. 1986;6:792-800.
102. Emmerman M, Temin HM. Comparison of promoter suppression in avian and murine retrovirus vectors. Nucleic Acids Res. 1986;14:9381-9396.
103. Clark EA, Golub TR, Lander ES, et al. Genomic analysis of metastasis reveals an essential role for RhoC. Nature. 2000;406:532-535.
104. Amatschek S, Koenig U, Auer H, et al. Tissue-wide expression profiling using cDNA subtraction and microarrays to identify tumor-specific genes. Cancer Res. 2004;64:844-856.
105. Hafkemeyer P, Brinkmann U, Gottesman, M, et al. Apoptosis induced by Pseudomonas exotoxin: a sensitive and rapid marker for gene delivery in vivo. Hum Gene Ther. 1999;10:923-934.
106. Brinkmann U, Pastan I. Immunotoxins against cancer. Biochim Biophs Acta. 1994;1198:27-45.
107. Martin V, Cortes ML de Felipe P, et al. Cancer gene therapy by thyroid-hormone mediated expression of toxin genes. Cancer. Res. 2000;60:218-3224.
108. Gossen M, Freundlieb S, Bender G, et al. Transcriptional activation by tetracyclines in mammalian cells. Science. 1995;268:1766-1769.
109. No D, Yao TP, Evans RM. Ecdysone-inducible gene expression in mammalian cells and transgenic mice. Proc. Natl Acad Sci USA. 1996;93:3346-3351.
110. Gupta S, Schoer R, Egan JE, et al. Inducible, reversible, and stable RNA interference in mammalian cells. Proc Natl Acad Sci USA. 2004;101:1927-1932.
111. Gupta S, Schoer R, Egan JE, et al. Inducible, reversible and stable RNA interference in mammalian cells. Proc Natl Acad Sci USA. 2004;101:1927-1932.
112. Van Craenenbroeck K, Vanhoenacker P, Leysen JE, et al. Evaluation of the tetracycline- and ecdysone-inducible systems for expression of neurotransmitter receptors in mammalian cells. Eur J Neurosci. 2001;14:968-976.
113. Izquierdo M, Cortés ML, de Felipe P, et al. Long-term rat survival after malignant brain tumor regression by retroviral gene therapy. Gene Ther. 1995;2:66-69.
114. Cortés ML, de Felipe P, Martin V, et al. Successful use of a plant gene in the treatment of cancer in vivo. Gene Ther. 1998;5:1499-1507.
115. Cortés ML, García-Escudero V, Hughes M, et al. The cyanide bystander effect of the linamarase/linamarin killer-suicide gene therapy system. J Gene Med. 2002;4:1-8.
116. Izquierdo M, Martin V, de Felipe P, et al. Human malignant brain tumor response to herpes simplex thymidine kinase (HSVtk)/ganciclovir gene therapy. Gene Ther. 1996;3:491-495.
117. Ram Z, Culver KW, Oshiro EM, et al. Therapy of malignant brain tumors by intratumoral implantation of retroviral vector-producing cells. Nat Med. 1997;3:1354-1361.
118. Brummelkamp TR, Bernards R. New tools for functional mammalian cancer genetics. Nat Rev Cancer. 2003;3:781-789.