MicroRNA silencing for cancer therapy targeted to the tumour microenvironment

Nature

Volumn 518, Issue 7537, 2015, Pages 107-110

  Cheng, Christopher J ^a,b,d   Bahal, Raman ^c   Babar, Imran A ^a,e   Pincus, Zachary ^a,f   Barrera, Francisco ^a,g   Liu, Connie ^a   Svoronos, Alexander ^a   Braddock, Demetrios T ^c   Glazer, Peter M ^c   Engelman, Donald M ^a   Saltzman, W Mark ^b   Slack, Frank J ^h  

^a YALE UNIVERSITY   (United States)

^b Yale University   (United States)

^c YALE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d ALEXION PHARMACEUTICALS   (United States)

^e OrbiMed Advisors LLC   (United States)

^f WASHINGTON UNIVERSITY   (United States)

^g UNIVERSITY OF TENNESSEE   (United States)

^h BETH ISRAEL DEACONESS MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTINEOPLASTIC AGENT; ANTISENSE OLIGOMER; ANTISENSE OLIGONUCLEOTIDE; EPIDERMAL GROWTH FACTOR RECEPTOR; EPIDERMAL GROWTH FACTOR RECEPTOR 2; MICRORNA; MICRORNA 155; MICRORNA 182; MICRORNA 21; MICRORNA 210; PEPTIDE; PEPTIDE NUCLEIC ACID; UNCLASSIFIED DRUG; ACID; MIRN155 MICRORNA, MOUSE; NANOPARTICLE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANTISENSE THERAPY; ARTICLE; CANCER THERAPY; CELL MEMBRANE; CLINICAL EFFECTIVENESS; CONTROLLED STUDY; DISEASE CLEARANCE; DRUG DELIVERY SYSTEM; DRUG EFFICACY; ENZYME INHIBITION; GENE EXPRESSION; GENE SILENCING; GENE TARGETING; IN VIVO STUDY; LYMPHOMA; MOUSE; NONHUMAN; ONCOGENE; PH; PHYSIOLOGICAL PROCESS; PRIORITY JOURNAL; RNA TRANSPORT; SOLID TUMOR; TUMOR MICROENVIRONMENT; ANIMAL; ANTAGONISTS AND INHIBITORS; CELL MEMBRANE PERMEABILITY; CHEMISTRY; DISEASE MODEL; FEMALE; GENE EXPRESSION REGULATION; GENETICS; MALE; METABOLISM; MOLECULARLY TARGETED THERAPY; PATHOLOGY;

ACIDS; ANIMALS; CELL MEMBRANE; CELL MEMBRANE PERMEABILITY; DISEASE MODELS, ANIMAL; DRUG DELIVERY SYSTEMS; FEMALE; GENE EXPRESSION REGULATION, NEOPLASTIC; GENE SILENCING; HYDROGEN-ION CONCENTRATION; LYMPHOMA; MALE; MICE; MICRORNAS; MOLECULAR TARGETED THERAPY; NANOPARTICLES; ONCOGENES; PEPTIDE NUCLEIC ACIDS; TUMOR MICROENVIRONMENT;

EID: 84924424616     PISSN: 00280836     EISSN: 14764687     Source Type: Journal    
DOI: 10.1038/nature13905     Document Type: Article

References (37)

1. He, L. & Hannon, G. J. MicroRNAs: small RNAs with a big role in gene regulation. Nature Rev. Genet. 5, 522-531 (2004).
2. Calin, G. A. & Croce, C. M. MicroRNA signatures in human cancers. Nature Rev. Cancer 6, 857-866 (2006).
3. Esquela-Kerscher, A. & Slack, F. J. Oncomirs - microRNAs with a role in cancer. Nature Rev. Cancer 6, 259-269 (2006).
4. Medina, P. P., Nolde, M. & Slack, F. J. OncomiR addiction in an in vivo model of microRNA-21-induced pre-B-cell lymphoma. Nature 467, 86-90 (2010).
5. Babar, I. A. et al. Nanoparticle-based therapy in an in vivo microRNA-155 (miR-155)-dependent mouse model of lymphoma. Proc. Natl Acad. Sci. USA 109, E1695-E1704 (2012).
6. Stenvang, J., Petri, A., Lindow, M., Obad, S. & Kauppinen, S. Inhibition of microRNA function by antimiR oligonucleotides. Silence 3, 1 (2012).
7. Kasinski, A. L. & Slack, F. J. Epigenetics and genetics. MicroRNAs en route to the clinic: progress in validating and targeting microRNAs for cancer therapy. Nature Rev. Cancer 11, 849-864 (2011).
8. Cheng, C. J., Saltzman, W. M. & Slack, F. J. Canonical and non-canonical barriers facing antimiR cancer therapeutics. Curr. Med. Chem. 20, 3582-3593 (2013).
9. Elmen, J. et al. LNA-mediated microRNA silencing in non-human primates. Nature 452, 896-899 (2008).
10. Krutzfeldt, J. et al. Silencing of microRNAs in vivo with 'antagomirs'. Nature 438, 685-689 (2005).
11. White, P. J., Anastasopoulos, F., Pouton, C. W. & Boyd, B. J. Overcoming biological barriers to in vivo efficacy of antisense oligonucleotides. Expert Rev. Mol. Med. 11, e10 (2009).
12. Vaupel, P., Kallinowski, F. & Okunieff, P. Blood flow, oxygen and nutrient supply, and metabolic microenvironment of human tumors: a review. Cancer Res. 49, 6449-6465 (1989).
13. Reshetnyak, Y. K., Andreev, O. A., Lehnert, U. & Engelman, D. M. Translocation of molecules into cells by pH-dependent insertion of a transmembrane helix. Proc. Natl Acad. Sci. USA 103, 6460-6465 (2006).
14. Thevenin, D., An, M. & Engelman, D. M. pHLIP-mediated translocation of membrane impermeable molecules into cells. Chem. Biol. 16, 754-762 (2009).
15. Reshetnyak, Y. K. et al. Measuring tumor aggressiveness and targeting metastatic lesions with fluorescent pHLIP. Mol. Imaging Biol. 13, 1146-1156 (2011).
16. Nielsen, P. E., Egholm, M. & Buchardt, O. Peptide nucleic acid (PNA). A DNA mimic with a peptide backbone. Bioconjug. Chem. 5, 3-7 (1994).
17. Østergaard, H., Tachibana, C. & Winther, J. R. Monitoring disulfide bond formation in the eukaryotic cytosol. J. Cell Biol. 166, 337-345 (2004).
18. Rai, D., Karanti, S., Jung, I., Dahia, P. L. M. & Aguiar, R. C. T. Coordinated expression of microRNA-155 and predicted target genes in diffuse large B-cell lymphoma. Cancer Genet. Cytogenet. 181, 8-15 (2008).
19. Rather, M. I., Nagashri, M. N., Swamy, S. S., Gopinath, K. S. & Kumar, A. Oncogenic microRNA-155 down-regulates tumor suppressor CDC73 and promotes oral squamous cell carcinoma cell proliferation: implications for cancer therapeutics. J. Biol. Chem. 288, 608-618 (2013).
20. Costinean, S. et al. Pre-B cell proliferation and lymphoblastic leukemia/high-grade lymphoma in Em-miR155 transgenic mice. Proc. Natl Acad. Sci. USA 103, 7024-7029 (2006).
21. Sandhu, S. K., Croce, C. M. & Garzon, R. Micro-RNA expression and function in lymphomas. Adv. Hematol. 2011, 1-12 (2011).
22. Cheng, C. J.& Slack, F. J. The duality of oncomiR addiction in the maintenance and treatment of cancer. Cancer J. 18, 232-237 (2012).
23. DeVita, V. T., Lawrence, T. S. & Rosenberg, S. A. DeVita, Hellman, and Rosenberg's Cancer: Principles & Practice of Oncology 9th edn (Lippincott Williams & Wilkins, 2011).
24. Zhang, Y. et al. LNA-mediated anti-miR-155 silencing in low-grade B-cell lymphomas. Blood 120, 1678-1686 (2012).
25. Gottwein, E. et al. A viral microRNA functions as an orthologue of cellular miR-155. Nature 450, 1096-1099 (2007).
26. Dweep, H., Sticht, C., Pandey, P. & Gretz, N. miRWalk-database: prediction of possible miRNA binding sites by 'walking' the genes of three genomes. J. Biomed. Inform. 44, 839-847 (2011).
27. Li, S. et al. microRNA-155 silencing inhibits proliferation and migration and induces apoptosis by upregulating BACH1 in renal cancer cells. Mol. Med. Report. 5, 949-954 (2012).
28. Brognara, E. et al. Uptake by human glioma cell lines and biological effects of a peptide-nucleic acids targeting miR-221. J. Neurooncol. 118, 19-28 (2014).
29. Wang, Y.-Z. et al. Delivery of an miR155 inhibitor by anti-CD20 single-chain antibody into B cells reduces the acetylcholine receptor-specific autoantibodies andameliorates experimental autoimmunemyasthenia gravis. Clin. Exp. Immunol. 176, 207-221 (2014).
30. Christensen, L. et al. Solid-phase synthesis of peptide nucleic acids. J. Pept. Sci. 1, 175-183 (1995).
31. Obad, S. et al. Silencing of microRNAfamilies by seed-targeting tiny LNAs. Nature Genet. 43, 371-378 (2011).
32. Sahu, B. et al. Synthesis and characterization of conformationally preorganized, (R)-diethylene glycol-containing c-peptide nucleic acids with superior hybridization properties and water solubility. J. Org. Chem. 76, 5614-5627 (2011).
33. Fabani, M. M. et al. Efficient inhibition of miR-155 function in vivo by peptide nucleic acids. Nucleic Acids Res. 38, 4466-4475 (2010).
34. Shiraishi, T. & Nielsen, P. E. Peptide nucleic acid (PNA) cell penetrating peptide (CPP) conjugates as carriers for cellular delivery of antisense oligomers. Artif. DNA PNA XNA 2, 90-99 (2011).
35. Fabbri, E. et al. miRNA therapeutics: delivery and biological activity of peptide nucleic acids targeting miRNAs. Epigenomics 3, 733-745 (2011).
36. Xu, G. et al. Transcriptome and targetome analysis in MIR155 expressing cells using RNA-seq. RNA 16, 1610-1622 (2010).
37. Loeb, G. B. et al. Transcriptome-wide miR-155 binding map reveals widespread noncanonical microRNA targeting. Mol. Cell 48, 760-770 (2012).