Knockdown of β-catenin with dicer-substrate siRNAs reduces liver tumor burden in vivo

Molecular Therapy

Volumn 22, Issue 1, 2014, Pages 92-101

  Dudek, Henryk ^a   Wong, Darren H ^a,f   Arvan, Rokhand ^a   Shah, Anee ^a   Wortham, Kathleen ^a   Ying, Bo ^a   Diwanji, Rohan ^a   Zhou, Wei ^a   Holmes, Benjamin ^a   Yang, Hailin ^a   Cyr, Wendy A ^a   Zhou, Yi ^a   Shah, Aalok ^a   Farkiwala, Ruchir ^a   Lee, Michael ^a,d   Li, Yiting ^a,e   Rettig, Garrett R ^b   Collingwood, Michael A ^b   Basu, Sujit K ^a,c   Behlke, Mark A ^b   Brown, Bob D ^a   more..

^a Dicerna Pharmaceuticals   (United States)

^b INTEGRATED DNA TECHNOLOGIES   (United States)

^c Shire Human Genetic Therapies   (United States)

^d Acceleron Pharma   (United States)

^e NORTHEASTERN UNIVERSITY   (United States)

^f Medivation Inc   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA CATENIN; DICER SUBSTRATE SHORT INTERFERING RNA; MESSENGER RNA; SMALL INTERFERING RNA; SOLID LIPID NANOPARTICLE; SORAFENIB; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CANCER INHIBITION; CONTROLLED STUDY; DRUG ACTIVITY; DRUG DELIVERY SYSTEM; DRUG EFFICACY; DRUG TISSUE LEVEL; GENE EXPRESSION; GENE SILENCING; HUMAN; HUMAN CELL; IMMUNOSTIMULATION; IN VIVO STUDY; LIVER TUMOR; MALE; MOUSE; NONHUMAN; PROTEIN EXPRESSION; PROTEIN TARGETING; RNA INTERFERENCE; TUMOR VOLUME;

ANIMALS; BETA CATENIN; CELL LINE, TUMOR; DISEASE MODELS, ANIMAL; GENE EXPRESSION; GENE KNOCKDOWN TECHNIQUES; HUMANS; LIVER NEOPLASMS; MALE; MICE; NANOPARTICLES; RIBONUCLEASE III; RNA INTERFERENCE; RNA, SMALL INTERFERING; TUMOR BURDEN; XENOGRAFT MODEL ANTITUMOR ASSAYS;

EID: 84891831905     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.233     Document Type: Article

References (43)

1. Traxler, P, Bold, G, Buchdunger, E, Caravatti, G, Furet, P, Manley, P et al. (2001). Tyrosine kinase inhibitors: From rational design to clinical trials. Med Res Rev 21: 499-512
2. Minna, JD and Dowell, J (2005). Erlotinib hydrochloride. Nat Rev Drug Discov (suppl): S14-S15
3. Bollag, G, Freeman, S, Lyons, JF and Post, LE (2003). Raf pathway inhibitors in oncology. Curr Opin Investig Drugs 4: 1436-1441
4. Williams, JA, Guicherit, OM, Zaharian, BI, Xu, Y, Chai, L, Wichterle, H et al. (2003). Identification of a small molecule inhibitor of the hedgehog signaling pathway: Effects on basal cell carcinoma-like lesions. Proc Natl Acad Sci USA 100: 4616-4621
5. Tran, C, Ouk, S, Clegg, NJ, Chen, Y, Watson, PA, Arora, V et al. (2009). Development of a second-generation antiandrogen for treatment of advanced prostate cancer. Science 324: 787-790
6. Koehler, AN (2010). A complex task? Direct modulation of transcription factors with small molecules. Curr Opin Chem Biol 14: 331-340
7. Hanahan, D and Weinberg, RA (2011). Hallmarks of cancer: The next generation. Cell 144: 646-674
8. Polakis, P (2012). Drugging Wnt signalling in cancer. EMBO J 31: 2737-2746
9. Polakis, P (2012). Wnt signaling in cancer. Cold Spring Harb Perspect Biol 4: 1-13
10. Lessene, G, Czabotar, PE and Colman, PM (2008). BCL-2 family antagonists for cancer therapy. Nat Rev Drug Discov 7: 989-1000
11. Semenza, GL (2003). Targeting HIF-1 for cancer therapy. Nat Rev Cancer 3: 721-732
12. Dang, CV (2012). MYC on the path to cancer. Cell 149: 22-35
13. Darnell, JE Jr (2002). Transcription factors as targets for cancer therapy. Nat Rev Cancer 2: 740-749
14. Rettig, GR and Behlke, MA (2012). Progress toward in vivo use of siRNAs-II. Mol Ther 20: 483-512
15. Sashital, DG and Doudna, JA (2010). Structural insights into RNA interference. Curr Opin Struct Biol 20: 90-97
16. Chua, JH, Armugam, A and Jeyaseelan, K (2009). MicroRNAs: Biogenesis, function and applications. Curr Opin Mol Ther 11: 189-199
17. Castanotto, D and Rossi, JJ (2009). The promises and pitfalls of RNA-interferencebased therapeutics. Nature 457: 426-433
18. Vaishnaw, AK, Gollob, J, Gamba-Vitalo, C, Hutabarat, R, Sah, D, Meyers, R et al. (2010). A status report on RNAi therapeutics. Silence 1: 14
19. Wang, Z, Rao, DD, Senzer, N and Nemunaitis, J (2011). RNA interference and cancer therapy. Pharm Res 28: 2983-2995
20. Burnett, JC, Rossi, JJ and Tiemann, K (2011). Current progress of siRNA/shRNA therapeutics in clinical trials. Biotechnol J 6: 1130-1146
21. Kim, DH, Behlke, MA, Rose, SD, Chang, MS, Choi, S and Rossi, JJ (2005). Synthetic dsRNA Dicer substrates enhance RNAi potency and efficacy. Nat Biotechnol 23: 222-226
22. Amarzguioui, M, Lundberg, P, Cantin, E, Hagstrom, J, Behlke, MA and Rossi, JJ (2006). Rational design and in vitro and in vivo delivery of Dicer substrate siRNA. Nat Protoc 1: 508-517
23. Zhou, J, Song, MS, Jacobi, AM, Behlke, MA, Wu, X and Rossi, JJ (2012). Deep sequencing analyses of DsiRNAs reveal the influence of 3' terminal overhangs on dicing polarity, strand selectivity, and RNA editing of siRNAs. Mol Ther Nucleic Acids 1: E17
24. Barker, N and Clevers, H (2006). Mining the Wnt pathway for cancer therapeutics. Nat Rev Drug Discov 5: 997-1014
25. Guichard, C, Amaddeo, G, Imbeaud, S, Ladeiro, Y, Pelletier, L, Maad, IB et al. (2012). Integrated analysis of somatic mutations and focal copy-number changes identifies key genes and pathways in hepatocellular carcinoma. Nat Genet 44: 694-698
26. Boyault, S, Rickman, DS, de Reyniès, A, Balabaud, C, Rebouissou, S, Jeannot, E et al. (2007). Transcriptome classification of HCC is related to gene alterations and to new therapeutic targets. Hepatology 45: 42-52
27. Zucman-Rossi, J and Laurent-Puig, P (2007). Genetic diversity of hepatocellular carcinomas and its potential impact on targeted therapies. Pharmacogenomics 8: 997-1003
28. Teufel, A, Staib, F, Kanzler, S, Weinmann, A, Schulze-Bergkamen, H and Galle, PR (2007). Genetics of hepatocellular carcinoma. World J Gastroenterol 13: 2271-2282
29. Tward, AD, Jones, KD, Yant, S, Cheung, ST, Fan, ST, Chen, X et al. (2007). Distinct pathways of genomic progression to benign and malignant tumors of the liver. Proc Natl Acad Sci USA 104: 14771-14776
30. But, DY, Lai, CL and Yuen, MF (2008). Natural history of hepatitis-related hepatocellular carcinoma. World J Gastroenterol 14: 1652-1656
31. O'Neil, BH and Venook, AP (2007). Hepatocellular carcinoma: The role of the North American GI Steering Committee Hepatobiliary Task Force and the advent of effective drug therapy. Oncologist 12: 1425-1432
32. Ma, WW and Hidalgo, M (2007). Exploiting novel molecular targets in gastrointestinal cancers. World J Gastroenterol 13: 5845-5856
33. Llovet, JM, Ricci, S, Mazzaferro, V, Hilgard, P, Gane, E, Blanc, JF et al.; SHARP Investigators Study Group. (2008). Sorafenib in advanced hepatocellular carcinoma. N Engl J Med 359: 378-390
34. Rose, SD, Kim, DH, Amarzguioui, M, Heidel, JD, Collingwood, MA, Davis, ME et al. (2005). Functional polarity is introduced by Dicer processing of short substrate RNAs. Nucleic Acids Res 33: 4140-4156
35. Jackson, AL and Linsley, PS (2010). Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application. Nat Rev Drug Discov 9: 57-67
36. Collingwood, MA, Rose, SD, Huang, L, Hillier, C, Amarzguioui, M, Wiiger, MT et al. (2008). Chemical modification patterns compatible with high potency dicer-substrate small interfering RNAs. Oligonucleotides 18: 187-200
37. Morrissey, DV, Lockridge, JA, Shaw, L, Blanchard, K, Jensen, K, Breen, W et al. (2005). Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs. Nat Biotechnol 23: 1002-1007
38. Tagami, T, Nakamura, K, Shimizu, T, Yamazaki, N, Ishida, T and Kiwada, H (2010). CpG motifs in pDNA-sequences increase anti-PEG IgM production induced by PEGcoated pDNA-lipoplexes. J Control Release 142: 160-166
39. Ma, Z, Li, J, He, F, Wilson, A, Pitt, B and Li, S (2005). Cationic lipids enhance siRNAmediated interferon response in mice. Biochem Biophys Res Commun 330: 755-759
40. Jho, EH, Zhang, T, Domon, C, Joo, CK, Freund, JN and Costantini, F (2002). Wnt/ beta-catenin/Tcf signaling induces the transcription of Axin2, a negative regulator of the signaling pathway. Mol Cell Biol 22: 1172-1183
41. Judge, AD, Robbins, M, Tavakoli, I, Levi, J, Hu, L, Fronda, A et al. (2009). Confirming the RNAi-mediated mechanism of action of siRNA-based cancer therapeutics in mice. J Clin Invest 119: 661-673
42. He, TC, Sparks, AB, Rago, C, Hermeking, H, Zawel, L, da Costa, LT et al. (1998). Identification of c-MYC as a target of the APC pathway. Science 281: 1509-1512
43. Foster, DJ, Barros, S, Duncan, R, Shaikh, S, Cantley, W, Dell, A et al. (2012). Comprehensive evaluation of canonical versus Dicer-substrate siRNA in vitro and in vivo. RNA 18: 557-568