RNA interference as therapeutics for hepatocellular carcinoma

Recent Patents on Anti-Cancer Drug Discovery

Volumn 6, Issue 1, 2011, Pages 106-115

  Xu, Chuanrui ^a,b,c   Lee, Susie A ^a   Chen, Xin ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b Union Hospital   (United States)

^c HUAZHONG UNIVERSITY OF SCIENCE AND TECHNOLOGY   (China)

Author keywords

Hcc; Rnai

Indexed keywords

ALNVSP 02; ANTINEOPLASTIC AGENT; ATU 027; BMI1 PROTEIN; CALAA 01; CYCLIN D1; CYCLIN E; HYPOXIA INDUCIBLE FACTOR; MACROGOL; MIRAVIRSEN; SHORT HAIRPIN RNA; SMALL INTERFERING RNA; STAT3 PROTEIN; SURVIVIN; TRANSCRIPTION FACTOR EZH2; TRANSFORMING GROWTH FACTOR BETA; UNCLASSIFIED DRUG; VASCULOTROPIN INHIBITOR; VIRUS VECTOR;

ADVANCED CANCER; ANTIANGIOGENIC ACTIVITY; APOPTOSIS; ARTICLE; CANCER INHIBITION; CELL CYCLE REGULATION; DRUG SAFETY; DRUG TARGETING; GENE SILENCING; GENE TARGETING; HEPATITIS; HEPATITIS B; HEPATITIS C; HUMAN; LIVER CELL CARCINOMA; LIVER FIBROSIS; LIVER TUMOR; MAMMAL CELL; MELANOMA; METASTASIS; MOLECULAR DYNAMICS; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PRIORITY JOURNAL; RNA INTERFERENCE; SIGNAL TRANSDUCTION; SOLID TUMOR; VIRUS ENTRY; VIRUS GENOME; VIRUS INHIBITION; VIRUS REPLICATION;

ANIMALS; CARCINOMA, HEPATOCELLULAR; GENE EXPRESSION REGULATION, NEOPLASTIC; GENE THERAPY; HUMANS; LIVER NEOPLASMS; MODELS, BIOLOGICAL; RNA INTERFERENCE; RNA, SMALL INTERFERING;

EID: 78650931075     PISSN: 15748928     EISSN: None     Source Type: Journal    
DOI: 10.2174/157489211793980097     Document Type: Article

References (100)

1. Parkin DM, Bray F, Ferlay J, Pisani P. Global cancer statistics, 2002. CA Cancer J Clin 2005; 55(2): 74-108
2. Schutte K, Bornschein J, Malfertheiner P. Hepatocellular carcinoma-epidemiological trends and risk factors. Dig Dis 2009; 27(2): 80-92
3. Kremsdorf D, Soussan P, Paterlini-Brechot P, Brechot C. Hepatitis B virus-related hepatocellular carcinoma: Paradigms for viralrelated human carcinogenesis. Oncogene 2006; 25(27): 3823-33
4. Bartosch B, Thimme R, Blum HE, Zoulim F. Hepatitis C virusinduced hepatocarcinogenesis. J Hepatol 2009; 51(4): 810-20
5. Taniguchi K, Roberts LR, Aderca IN, Dong X, Qian C, Murphy LM, et al. Mutational spectrum of beta-catenin, AXIN1, and AXIN2 in hepatocellular carcinomas and hepatoblastomas. Oncogene 2002; 21(31): 4863-71
6. Xu CR, Lee S, Ho C, Bommi P, Huang SA, Cheung ST, et al. Bmi1 functions as an oncogene independent of Ink4A/Arf repression in hepatic carcinogenesis. Mol Cancer Res 2009; 7(12): 1937-45
7. Huang GW, Yang LY, Lu WQ. Expression of hypoxia-inducible factor 1alpha and vascular endothelial growth factor in hepatocellular carcinoma: Impact on neovascularization and survival. World J Gastroenterol 2005; 11(11): 1705-8
8. Calvisi DF, Ladu S, Gorden A, Farina M, Conner EA, Lee JS, et al. Ubiquitous activation of Ras and Jak/Stat pathways in human HCC. Gastroenterology 2006; 130(4): 1117-28
9. Tward AD, Jones KD, Yant S, Cheung ST, Fan ST, Chen X, et al. Distinct pathways of genomic progression to benign and malignant tumors of the liver. Proc Natl Acad Sci USA 2007; 104(37): 14771-6
10. Harada N, Oshima H, Katoh M, Tamai Y, Oshima M, Taketo MM. Hepatocarcinogenesis in mice with beta-catenin and Ha-Ras gene mutations. Cancer Res 2004; 64(1): 48-54
11. Tanaka H, Yamamoto M, Hashimoto N, Miyakoshi M, Tamakawa S, Yoshie M, et al. Hypoxia-independent overexpression of hypoxia-inducible factor 1alpha as an early change in mouse hepatocarcinogenesis. Cancer Res 2006; 66(23): 11263-70
12. Lau WY, Lai EC, Hepatocellular carcinoma: Current management and recent advances. Hepatobiliary Pancreat Dis Int 2008; 7(3): 237-57
13. Llovet JM, Ricci S, Mazzaferro V, Hilgard P, Gane E, Blanc JF, et al. Sorafenib in advanced hepatocellular carcinoma. N Engl J Med 2008; 359(4): 378-90
14. 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; 391(6669): 806-11
15. Lau NC, Lim LP, Weinstein EG, Bartel DP. An abundant class of tiny RNAs with probable regulatory roles in Caenorhabditis elegans. Science 2001; 294(5543): 858-62
16. 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(6836): 494-8
17. Brummelkamp TR, Bernards R, Agami R. A system for stable expression of short interfering RNAs in mammalian cells. Science 2002; 296(5567): 550-3
18. Hamilton AJ, Baulcombe DC. A species of small antisense RNA in posttranscriptional gene silencing in plants. Science 1999; 286(5441): 950-2
19. Zamore PD, Tuschl T, Sharp PA, Bartel DP. RNAi: Doublestranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals. Cell 2000; 101(1): 25-33
20. Jinek M, Doudna JA. A three-dimensional view of the molecular machinery of RNA interference. Nature 2009; 457(7228): 405-12
21. Lee Y, Ahn C, Han J, Choi H, Kim J, Yim J, et al. The nuclear RNase III Drosha initiates microRNA processing. Nature 2003; 425(6956): 415-9
22. Han J, Lee Y, Yeom KH, Kim YK, Jin H, Kim VN. The Drosha- DGCR8 complex in primary microRNA processing. Genes Dev 2004; 18(24): 3016-27
23. Gregory RI, Yan KP, Amuthan G, Chendrimada T, Doratotaj B, Cooch N, et al. The Microprocessor complex mediates the genesis of microRNAs. Nature 2004; 432(7014): 235-40
24. Bernstein E, Caudy AA, Hammond SM, Hannon GJ. Role for a bidentate ribonuclease in the initiation step of RNA interference. Nature 2001; 409(6818): 363-6
25. Liu J, Carmell MA, Rivas FV, Marsden CG, Thomson JM, Song JJ, et al. Argonaute2 is the catalytic engine of mammalian RNAi. Science 2004; 305(5689): 1437-41
26. Tolia NH, Joshua-Tor L. Slicer and the argonautes. Nat Chem Biol 2007; 3(1): 36-43
27. Gregory RI, Chendrimada TP, Cooch N, Shiekhattar R. Human RISC couples microRNA biogenesis and posttranscriptional gene silencing. Cell 2005; 123(4): 631-40
28. Romano PR, McCallus DE, Pachuk CJ. RNA interferencemediated prevention and therapy for hepatocellular carcinoma. Oncogene 2006; 25(27): 3857-65
29. Rand TA, Petersen S, Du F, Wang X. Argonaute2 cleaves the antiguide strand of siRNA during RISC activation. Cell 2005; 123(4): 621-9
30. Hammond SM, Boettcher S, Caudy AA, Kobayashi R, Hannon GJ. Argonaute2, a link between genetic and biochemical analyses of RNAi. Science 2001; 293(5532): 1146-50
31. Matzke MA, Birchler JA. RNAi-mediated pathways in the nucleus. Nat Rev Genet 2005; 6(1): 24-35
32. Wassenegger M. The role of the RNAi machinery in heterochromatin formation. Cell 2005; 122(1): 13-6
33. Castanotto D, Rossi JJ. The promises and pitfalls of RNAinterference-based therapeutics. Nature 2009; 457(7228): 426-33
34. McCaffrey AP, Meuse L, Pham TT, Conklin DS, Hannon GJ, Kay MA. RNA interference in adult mice. Nature 2002; 418(6893): 38-9
35. Song E, Lee SK, Wang J, Ince N, Ouyang N, Min J, et al. RNA interference targeting Fas protects mice from fulminant hepatitis. Nat Med 2003; 9(3): 347-51
36. Zhang R, Ma L, Zheng M, Ren J, Wang T, Meng Y, et al. Survivin knockdown by short hairpin RNA abrogates the growth of human hepatocellular carcinoma xenografts in nude mice. Cancer Gene Ther 2010;17(4): 275-88
37. Li J, Piao YF, Jiang Z, Chen L, Sun HB. Silencing of signal transducer and activator of transcription 3 expression by RNA interference suppresses growth of human hepatocellular carcinoma in tumor-bearing nude mice. World J Gastroenterol 2009; 15(21): 2602-8
38. Brummelkamp TR, Bernards R, Agami R. Stable suppression of tumorigenicity by virus-mediated RNA interference. Cancer Cell 2002; 2(3): 243-7
39. 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(14): 3919-22
40. Jia RB, Zhang P, Zhou YX, Song X, Liu HY, Wang LZ, et al. VEGF-targeted RNA interference suppresses angiogenesis and tumor growth of retinoblastoma. Ophthalmic Res 2007; 39(2): 108-15
41. Thorgeirsson SS, Grisham JW. Molecular pathogenesis of human hepatocellular carcinoma. Nat Genet 2002; 31(4): 339-46
42. Mason WS, Aldrich C, Summers J, Taylor JM. Asymmetric replication of duck hepatitis B virus DNA in liver cells: Free minus-strand DNA. Proc Natl Acad Sci USA 1982; 79(13): 3997-4001
43. Weiser B, Ganem D, Seeger C, Varmus HE. Closed circular viral DNA and asymmetrical heterogeneous forms in livers from animals infected with ground squirrel hepatitis virus. J Virol 1983; 48(1): 1-9
44. Grimm D, Kay MA. Therapeutic short hairpin RNA expression in the liver: viral targets and vectors. Gene Ther 2006; 13(6): 563-75
45. Farazi PA, DePinho RA. Hepatocellular carcinoma pathogenesis: From genes to environment. Nat Rev Cancer 2006; 6(9): 674-87
46. Chen Z, Xu ZF, Ye JJ, Yao HP, Zheng S, Ding JY. Combination of small interfering RNAs mediates greater inhibition of human hepatitis B virus replication and antigen expression. J Zhejiang Univ Sci B 2005; 6(4): 236-41
47. Zhang XN, Xiong W, Wang JD, Hu YW, Xiang L, Yuan ZH. siRNA-mediated inhibition of HBV replication and expression. World J Gastroenterol 2004; 10(20): 2967-71
48. Sun Y, Li Z, Li L, Li J, Liu X, Li W. Effective inhibition of hepatitis B virus replication by small interfering RNAs expressed from human foamy virus vectors. Int J Mol Med 2007; 19(4): 705-11
49. McCaffrey AP, Nakai H, Pandey K, Huang Z, Salazar FH, Xu H, et al. Inhibition of hepatitis B virus in mice by RNA interference. Nat Biotechnol 2003; 21(6): 639-44
50. Shlomai A, Shaul Y. Inhibition of hepatitis B virus expression and replication by RNA interference. Hepatology 2003; 37(4): 764-70
51. Ren XR, Zhou LJ, Luo GB, Lin B, Xu A. Inhibition of hepatitis B virus replication in 2.2.15 cells by expressed shRNA. J Viral Hepat 2005; 12(3): 236-42
52. Ren X, Luo G, Xie Z, Zhou L, Kong X, Xu A. Inhibition of multiple gene expression and virus replication of HBV by stable RNA interference in 2.2.15 cells. J Hepatol 2006; 44(4): 663-70
53. Carmona S, Ely A, Crowther C, Moolla N, Salazar FH, Marion PL, et al. Effective inhibition of HBV replication in vivo by anti-HBx short hairpin RNAs. Mol Ther 2006; 13(2): 411-21
54. Uprichard SL, Boyd B, Althage A, Chisari FV. Clearance of hepatitis B virus from the liver of transgenic mice by short hairpin RNAs. Proc Natl Acad Sci USA 2005; 102(3): 773-8
55. Kim YH, Lee JH, Paik NW, Rho HM. RNAi-based knockdown of HBx mRNA in HBx-transformed and HBV-producing human liver cells. DNA Cell Biol 2006; 25(7): 412-7
56. Deng L, Li G, Xi L, Yin A, Gao Y, You W, et al. Hepatitis B virus inhibition in mice by lentiviral vector mediated short hairpin RNA. BMC Gastroenterol 2009; 9: 73
57. Chen Y, Du D, Wu J, Chan CP, Tan Y, Kung HF, et al. Inhibition of hepatitis B virus replication by stably expressed shRNA. Biochem Biophys Res Commun 2003; 311(2): 398-404
58. Ni Q, Chen Z, Yao HP, Yang ZG, Liu KZ, Wu LL. Inhibition of human La protein by RNA interference downregulates hepatitis B virus mRNA in 2.2.15 cells. World J Gastroenterol 2004; 10(14): 2050-4
59. Kung, H.-F., He, M.-L. Inhibition of hepatitis B virus (HBV) replication by RNA interference. US7067249 (2006)
60. Kim, M., Shin, D., Kim, S. I., Park, M. Small interfering RNA and pharmaceutical composition for treatment of hepatitis B comprising the same. US20100063132 (2010)
61. Wilson JA, Jayasena S, Khvorova A, Sabatinos S, Rodrigue-Gervais IG, Arya S, et al. RNA interference blocks gene expression and RNA synthesis from hepatitis C replicons propagated in human liver cells. Proc Natl Acad Sci USA 2003; 100(5): 2783-8
62. Kronke J, Kittler R, Buchholz F, Windisch MP, Pietschmann T, Bartenschlager R, et al. Alternative approaches for efficient inhibition of hepatitis C virus RNA replication by small interfering RNAs. J Virol 2004; 78(7): 3436-46
63. Yokota T, Sakamoto N, Enomoto N, Tanabe Y, Miyagishi M, Maekawa S, et al. Inhibition of intracellular hepatitis C virus replication by synthetic and vector-derived small interfering RNAs. EMBO Rep 2003; 4(6): 602-8
64. Takigawa Y, Nagano-Fujii M, Deng L, Hidajat R, Tanaka M, Mizuta H, et al. Suppression of hepatitis C virus replicon by RNA interference directed against the NS3 and NS5B regions of the viral genome. Microbiol Immunol 2004; 48(8): 591-8
65. Wang Q, Contag CH, Ilves H, Johnston BH, Kaspar RL. Small hairpin RNAs efficiently inhibit hepatitis C IRES-mediated gene expression in human tissue culture cells and a mouse model. Mol Ther 2005; 12(3): 562-8
66. Wilson JA, Richardson CD. Hepatitis C virus replicons escape RNA interference induced by a short interfering RNA directed against the NS5b coding region. J Virol 2005; 79(11): 7050-8
67. Lindenbach BD, Evans MJ, Syder AJ, Wolk B, Tellinghuisen TL, Liu CC, et al. Complete replication of hepatitis C virus in cell culture. Science 2005; 309(5734): 623-6
68. Wakita T, Pietschmann T, Kato T, Date T, Miyamoto M, Zhao Z, et al. Production of infectious hepatitis C virus in tissue culture from a cloned viral genome. Nat Med 2005; 11(7): 791-6
69. Randall G, Panis M, Cooper JD, Tellinghuisen TL, Sukhodolets KE, Pfeffer S, et al. Cellular cofactors affecting hepatitis C virus infection and replication. Proc Natl Acad Sci USA 2007; 104(31): 12884-9
70. Bessard A, Fremin C, Ezan F, Fautrel A, Gailhouste L, Baffet G. RNAi-mediated ERK2 knockdown inhibits growth of tumor cells in vitro and in vivo. Oncogene 2008; 27(40): 5315-25
71. Lahsnig C, Mikula M, Petz M, Zulehner G, Schneller D, van Zijl F, et al. ILEI requires oncogenic Ras for the epithelial to mesenchymal transition of hepatocytes and liver carcinoma progression. Oncogene 2009; 28(5): 638-50
72. Sun HX, He HW, Zhang SH, Liu TG, Ren KH, He QY, et al. Suppression of N-Ras by shRNA-expressing plasmid increases sensitivity of HepG2 cells to vincristine-induced growth inhibition. Cancer Gene Ther 2009; 16(9): 693-702
73. Liu LX, Lee NP, Chan VW, Xue W, Zender L, Zhang C, et al. Targeting cadherin-17 inactivates Wnt signaling and inhibits tumor growth in liver carcinoma. Hepatology 2009; 50(5): 1453-63
74. Bramlage, B., Hossbach, M., Tan, P., Vornlocher, H.-P. Compositions and methods for inhibiting expression of TGF-beta receptor genes. WO2010006973 (2010)
75. Sudo T, Utsunomiya T, Mimori K, Nagahara H, Ogawa K, Inoue H, et al. Clinicopathological significance of EZH2 mRNA expression in patients with hepatocellular carcinoma. Br J Cancer 2005; 92(9): 1754-8
76. Chen Y, Lin MC, Yao H, Wang H, Zhang AQ, Yu J, et al. Lentivirus-mediated RNA interference targeting enhancer of zeste homolog 2 inhibits hepatocellular carcinoma growth through downregulation of stathmin. Hepatology 2007; 46(1): 200-8
77. Costa, R., Wang, I.-C. Methods of inhibiting tumor cell proliferation with FoxM1 siRNA. US20100098663 (2010)
78. Malumbres M, Barbacid M. To cycle or not to cycle: A critical decision in cancer. Nat Rev Cancer 2001; 1(3): 222-31
79. Li K, Lin SY, Brunicardi FC, Seu P. Use of RNA interference to target cyclin E-overexpressing hepatocellular carcinoma. Cancer Res 2003; 63(13): 3593-7
80. Macarulla T, Ramos FJ, Tabernero J. Aurora kinase family: A new target for anticancer drug. Recent Pat Anticancer Drug Discov 2008; 3(2): 114-22
81. Lapenna S, Giordano A. Cell cycle kinases as therapeutic targets for cancer. Nat Rev Drug Discov 2009; 8(7): 547-66
82. Addepalli, M. K., Reddy, V. E. C. G. RNA interference mediated inhibition of aurorakinase B and its combinations as anticancer therapy. US7615627 (2009)
83. Raskopf E, Vogt A, Sauerbruch T, Schmitz V. siRNA targeting VEGF inhibits hepatocellular carcinoma growth and tumor angiogenesis in vivo. J Hepatol 2008; 49(6): 977-84
84. Tolentino, M. J., Reich, S. J. Compositions and methods for siRNA inhibition of angiogenesis. US7345027 (2008)
85. Liu, Y., Lu, P. Y., Woodle, M. C., Xie, F. Y. Composition and methods of RNAi therapeutics for treatment of cancer and other neovascularization diseases. US7723316 (2010)
86. Takahashi Y, Nishikawa M, Takakura Y. Inhibition of tumor cell growth in the liver by RNA interference-mediated suppression of HIF-1alpha expression in tumor cells and hepatocytes. Gene Ther 2008; 15(8): 572-82
87. Reich, S. J., Surace, E. M., Tolentino, M. J. Compositions and methods for siRNA inhibition of HIF-1 alpha. US7521431 (2009)
88. Li X, Pan Y, Fan R, Jin H, Han S, Liu J, et al. Adenovirusdelivered CIAPIN1 small interfering RNA inhibits HCC growth in vitro and in vivo. Carcinogenesis 2008; 29(8): 1587-93
89. Ambrosini G, Adida C, Altieri DC. A novel anti-apoptosis gene, survivin, expressed in cancer and lymphoma. Nat Med 1997; 3(8): 917-21
90. Salvi A, Arici B, De Petro G, Barlati S. Small interfering RNA urokinase silencing inhibits invasion and migration of human hepatocellular carcinoma cells. Mol Cancer Ther 2004; 3(6): 671-8
91. Grimm D, Streetz KL, Jopling CL, Storm TA, Pandey K, Davis CR, et al. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 2006; 441(7092): 537-41
92. Sledz CA, Holko M, de Veer MJ, Silverman RH, Williams BR. Activation of the interferon system by short-interfering RNAs. Nat Cell Biol 2003; 5(9): 834-9
93. Agrawal S, Kandimalla ER. Role of Toll-like receptors in antisense and siRNA [corrected]. Nat Biotechnol 2004; 22(12): 1533-7
94. Jackson AL, Bartz SR, Schelter J, Kobayashi SV, Burchard J, Mao M, et al. Expression profiling reveals off-target gene regulation by RNAi. Nat Biotechnol 2003; 21(6): 635-7
95. Zimmermann TS, Lee AC, Akinc A, Bramlage B, Bumcrot D, Fedoruk MN, et al. RNAi-mediated gene silencing in non-human primates. Nature 2006; 441(7089): 111-4
96. Rozema DB, Lewis DL, Wakefield DH, Wong SC, Klein JJ, Roesch PL, et al. Dynamic PolyConjugates for targeted in vivo delivery of siRNA to hepatocytes. Proc Natl Acad Sci USA 2007; 104(32): 12982-7
97. Sato A, Takagi M, Shimamoto A, Kawakami S, Hashida M. Small interfering RNA delivery to the liver by intravenous administration of galactosylated cationic liposomes in mice. Biomaterials 2007; 28(7): 1434-42
98. Kim SI, Shin D, Choi TH, Lee JC, Cheon GJ, Kim KY, et al. Systemic and specific delivery of small interfering RNA's to the liver mediated by apolipoprotein A-I. Mol Ther 2007; 15(6): 1145-52
99. Akinc A, Zumbuehl A, Goldberg M, Leshchiner ES, Busini V, Hossain N, et al. A combinatorial library of lipid-like materials for delivery of RNAi therapeutics. Nat Biotechnol 2008; 26(5): 561-9
100. Nishina K, Unno T, Uno Y, Kubodera T, Kanouchi T, Mizusawa H, et al. Efficient in vivo delivery of siRNA to the liver by conjugation of alpha-tocopherol. Mol Ther 2008; 16(4): 734-40.