Development of siRNA payloads to target KRAS-Mutant cancer

Cancer Discovery

Volumn 4, Issue 10, 2014, Pages 1182-1197

  Yuan, Tina L ^a   Fellmann, Christof ^b   Lee, Chih Shia ^c   Ritchie, Cayde D ^a   Thapar, Vishal ^b,d   Lee, Liam C ^c   Hsu, Dennis J ^c   Grace, Danielle ^b,d   Carver, Joseph O ^c   Zuber, Johannes ^b,e   Luo, Ji ^c   McCormick, Frank ^a   Lowe, Scott W ^b,d,f  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b Howard Hughes Medical Institute Cold Spring Harbor Laboratory Cold Spring Harbor   (United States)

^c NATIONAL CANCER INSTITUTE   (United States)

^d MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

^e RESEARCH INSTITUTE OF MOLECULAR PATHOLOGY   (Austria)

^f HOWARD HUGHES MEDICAL INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

K RAS PROTEIN; NANOPARTICLE; RAF PROTEIN; SHORT HAIRPIN RNA; SMALL INTERFERING RNA; PHOSPHATIDYLINOSITOL 3 KINASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; APOPTOSIS; ARTICLE; CELL VIABILITY; COLORECTAL CANCER; CONTROLLED STUDY; DRUG EFFICACY; GENE EXPRESSION; GENE SILENCING; GROWTH INHIBITION; HUMAN; HUMAN CELL; IMMUNOHISTOCHEMISTRY; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; PROTEIN PHOSPHORYLATION; REAL TIME POLYMERASE CHAIN REACTION; RNA INTERFERENCE; SCORING SYSTEM; SENSOR; TUMOR GROWTH; WESTERN BLOTTING; ANIMAL; CLUSTER ANALYSIS; DISEASE MODEL; DRUG DELIVERY SYSTEM; DRUG SCREENING; GENE EXPRESSION PROFILING; GENE LIBRARY; GENE TRANSFER; GENETICS; METABOLISM; MUTATION; NEOPLASM; ONCOGENE RAS; PATHOLOGY; REPRODUCIBILITY; SIGNAL TRANSDUCTION; TUMOR CELL LINE; TUMOR VOLUME;

ANIMALS; CELL LINE, TUMOR; CLUSTER ANALYSIS; DISEASE MODELS, ANIMAL; DRUG DELIVERY SYSTEMS; GENE EXPRESSION PROFILING; GENE KNOCKDOWN TECHNIQUES; GENE LIBRARY; GENE TRANSFER TECHNIQUES; GENES, RAS; HUMANS; MICE; MUTATION; NANOPARTICLES; NEOPLASMS; PHOSPHATIDYLINOSITOL 3-KINASES; REPRODUCIBILITY OF RESULTS; RNA INTERFERENCE; RNA, SMALL INTERFERING; SIGNAL TRANSDUCTION; TUMOR BURDEN; XENOGRAFT MODEL ANTITUMOR ASSAYS;

EID: 84907528772     PISSN: 21598274     EISSN: 21598290     Source Type: Journal    
DOI: 10.1158/2159-8290.CD-13-0900     Document Type: Article

References (38)

1. Gysin S, Salt M, Young A, McCormick F. Therapeutic strategies for targeting ras proteins. Genes Cancer 2011; 2: 359-72.
2. Hatzivassiliou G, Song K, Yen I, Brandhuber BJ, Anderson DJ, Alvarado R, et al. RAF inhibitors prime wild-type RAF to activate the MAPK pathway and enhance growth. Nature 2010; 464: 431-5.
3. Poulikakos PI, Zhang C, Bollag G, Shokat KM, Rosen N. RAF inhibitors transactivate RAF dimers and ERK signalling in cells with wildtype BRAF. Nature 2010; 464: 427-30.
4. Rinehart J, Adjei AA, Lorusso PM, Waterhouse D, Hecht JR, Natale RB, et al. Multicenter phase II study of the oral MEK inhibitor, CI-1040, in patients with advanced non-small-cell lung, breast, colon, and pancreatic cancer. J Clin Oncol 2004; 22: 4456-62.
5. Haura EB, Ricart AD, Larson TG, Stella PJ, Bazhenova L, Miller VA, et al. A phase II study of PD-0325901, an oral MEK inhibitor, in previously treated patients with advanced non-small cell lung cancer. Clin Cancer Res 2010; 16: 2450-7.
6. Hainsworth JD, Cebotaru CL, Kanarev V, Ciuleanu TE, Damyanov D, Stella P, et al. A phase II, open-label, randomized study to assess the efficacy and safety of AZD6244 (ARRY-142886) versus pemetrexed in patients with non-small cell lung cancer who have failed one or two prior chemotherapeutic regimens. J Thorac Oncol 2010; 5: 1630-6.
7. Shimizu T, Tolcher AW, Papadopoulos KP, Beeram M, Rasco DW, Smith LS, et al. The clinical effect of the dual-targeting strategy involving PI3K/AKT/mTOR and RAS/MEK/ERK pathways in patients with advanced cancer. Clin Cancer Res 2012; 18: 2316-25.
8. Hannon GJ. RNA interference. Nature 2002; 418: 244-51.
9. Bartel DP. MicroRNAs: genomics, biogenesis, mechanism, and function. Cell 2004; 116: 281-97.
10. Hopkins AL, Groom CR. The druggable genome. Nat Rev Drug Discov 2002; 1: 727-30.
11. Patel MN, Halling-Brown MD, Tym JE, Workman P, Al-Lazikani B. Objective assessment of cancer genes for drug discovery. Nat Rev Drug Discov 2013; 12: 35-50.
12. Zhou J, Shum KT, Burnett JC, Rossi JJ. Nanoparticle-based delivery of RNAi therapeutics: progress and challenges. Pharmaceuticals 2013; 6: 85-107.
13. Fellmann C, Lowe SW. Stable RNA interference rules for silencing. Nat Cell Biol 2014; 16: 10-8.
14. Fellmann C, Zuber J, McJunkin K, Chang K, Malone CD, Dickins RA, et al. Functional identification of optimized RNAi triggers using a massively parallel sensor assay. Mol Cell 2011; 41: 733-46.
15. Vert JP, Foveau N, Lajaunie C, Vandenbrouck Y. An accurate and interpretable model for siRNA efficacy prediction. BMC Bioinformatics 2006; 7: 520.
16. Singh A, Greninger P, Rhodes D, Koopman L, Violette S, Bardeesy N, et al. A gene expression signature associated with “K-Ras addiction” reveals regulators of EMT and tumor cell survival. Cancer Cell 2009; 15: 489-500.
17. 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: 635-7.
18. Birmingham A, Anderson EM, Reynolds A, Ilsley-Tyree D, Leake D, Fedorov Y, et al. 3′ UTR seed matches, but not overall identity, are associated with RNAi off-targets. Nat Methods 2006; 3: 199-204.
19. Khan AA, Betel D, Miller ML, Sander C, Leslie CS, Marks DS. Transfection of small RNAs globally perturbs gene regulation by endogenous microRNAs. Nat Biotechnol 2009; 27: 549-55.
20. Haley B, Zamore PD. Kinetic analysis of the RNAi enzyme complex. Nat Struct Mol Biol 2004; 11: 599-606.
21. Livingstone LR, White A, Sprouse J, Livanos E, Jacks T, Tlsty TD. Altered cell cycle arrest and gene amplification potential accompany loss of wild-type p53. Cell 1992; 70: 923-35.
22. Tay Y, Kats L, Salmena L, Weiss D, Tan SM, Ala U, et al. Codingindependent regulation of the tumor suppressor PTEN by competing endogenous mRNAs. Cell 2011; 147: 344-57.
23. Drosten M, Dhawahir A, Sum EY, Urosevic J, Lechuga CG, Esteban LM, et al. Genetic analysis of Ras signalling pathways in cell proliferation, migration and survival. Embo J 2010; 29: 1091-104.
24. Blasco RB, Francoz S, Santamaria D, Canamero M, Dubus P, Charron J, et al. c-Raf, but not B-Raf, is essential for development of K-Ras oncogene-driven non-small cell lung carcinoma. Cancer Cell 2011; 19: 652-63.
25. Karreth FA, Frese KK, DeNicola GM, Baccarini M, Tuveson DA. C-Raf is required for the initiation of lung cancer by K-Ras(G12D). Cancer Discov 2011; 1: 128-36.
26. Heidorn SJ, Milagre C, Whittaker S, Nourry A, Niculescu-Duvas I, Dhomen N, et al. Kinase-dead BRAF and oncogenic RAS cooperate to drive tumor progression through CRAF. Cell 2010; 140: 209-21.
27. Freeman AK, Ritt DA, Morrison DK. Effects of Raf dimerization and its inhibition on normal and disease-associated Raf signaling. Molecular Cell 2013; 49: 751-8.
28. Holderfield M, Merritt H, Chan J, Wallroth M, Tandeske L, Zhai H, et al. RAF inhibitors activate the MAPK pathway by relieving inhibitory autophosphorylation. Cancer Cell 2013; 23: 594-602.
29. Davis ME. The first targeted delivery of siRNA in humans via a selfassembling, cyclodextrin polymer-based nanoparticle: from concept to clinic. Mol Pharm 2009; 6: 659-68.
30. Davis ME, Zuckerman JE, Choi CH, Seligson D, Tolcher A, Alabi CA, et al. Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nature 2010; 464: 1067-70.
31. Hu-Lieskovan S, Heidel JD, Bartlett DW, Davis ME, Triche TJ. Sequence-specific knockdown of EWS-FLI1 by targeted, nonviral delivery of small interfering RNA inhibits tumor growth in a murine model of metastatic Ewing’s sarcoma. Cancer Res 2005; 65: 8984-92.
32. Heidel JD, Yu Z, Liu JY, Rele SM, Liang Y, Zeidan RK, et al. Administration in non-human primates of escalating intravenous doses of targeted nanoparticles containing ribonucleotide reductase subunit M2 siRNA. Proc Natl Acad Sci U S A 2007; 104: 5715-21.
33. Castellano E, Sheridan C, Thin MZ, Nye E, Spencer-Dene B, Diefenbacher ME, et al. Requirement for interaction of PI3-kinase p110alpha with RAS in lung tumor maintenance. Cancer Cell 2013; 24: 617-30.
34. Gupta S, Ramjaun AR, Haiko P, Wang Y, Warne PH, Nicke B, et al. Binding of ras to phosphoinositide 3-kinase p110alpha is required for ras-driven tumorigenesis in mice. Cell 2007; 129: 957-68.
35. Xue W, Dahlman JE, Tammela T, Khan OF, Sood S, Dave A, et al. Small RNA combination therapy for lung cancer. Proc Natl Acad Sci U S A 2014;111:E3553–61.
36. Karnoub AE, Weinberg RA. Ras oncogenes: split personalities. Nat Rev Mol Cell Biol 2008; 9: 517-31.
37. Weng MT, Lee JH, Wei SC, Li Q, Shahamatdar S, Hsu D, et al. Evolutionarily conserved protein ERH controls CENP-E mRNA splicing and is required for the survival of KRAS mutant cancer cells. Proc Natl Acad Sci U S A 2012; 109: E3659-67.
38. Dickins RA, Hemann MT, Zilfou JT, Simpson DR, Ibarra I, Hannon GJ, et al. Probing tumor phenotypes using stable and regulated synthetic microRNA precursors. Nat Genet 2005; 37: 1289-95.