Targeting MicroRNAs with small molecules: From dream to reality

Clinical Pharmacology and Therapeutics

Volumn 87, Issue 6, 2010, Pages 754-758

  Zhang, S ^a   Chen, L ^a   Jung, E J ^a   Calin, G A ^a  

^a UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTAGOMIR; ANTISENSE OLIGONUCLEOTIDE; DEOXYRIBONUCLEASE; LOCKED NUCLEIC ACID; MICRORNA; OLIGONUCLEOTIDE; RIBOZYME; SHORT HAIRPIN RNA; SMALL INTERFERING RNA; SMALL MOLECULE TRANSPORT AGENT; UNCLASSIFIED DRUG;

APOPTOSIS; CANCER CELL; CANCER THERAPY; CELL DIFFERENTIATION; CELL PROLIFERATION; CHRONIC LYMPHATIC LEUKEMIA; CLINICAL TRIAL; COMPUTER MODEL; DRUG DESIGN; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENE EXPRESSION PROFILING; GENE OVEREXPRESSION; GENE TARGETING; HUMAN; MOLECULAR GENETICS; MOLECULE; NEOPLASM; PATHOGENESIS; PRIORITY JOURNAL; PROGNOSIS; PROTEIN FUNCTION; REVIEW; TREATMENT RESPONSE;

ANIMALS; ANTINEOPLASTIC AGENTS; DRUG DELIVERY SYSTEMS; DRUG DESIGN; GENE EXPRESSION REGULATION, NEOPLASTIC; HUMANS; MICRORNAS; NEOPLASMS; UNITED STATES;

EID: 77952584924     PISSN: 00099236     EISSN: 15326535     Source Type: Journal    
DOI: 10.1038/clpt.2010.46     Document Type: Review

References (29)

1. Ambros, V. The evolution of our thinking about microRNAs. Nat. Med. 14, 1036-1040 (2008).
2. Ambros, V. & Chen, X. The regulation of genes and genomes by small RNAs. Development 134, 1635-1641 (2007).
3. Vasudevan, S., Tong, Y. & Steitz, J.A. Switching from repression to activation: microRNAs can up-regulate translation. Science 318, 1931-1934 (2007).
4. Calin, G.A. et al. MiR-15a and miR-16-1 cluster functions in human leukemia. Proc. Natl. Acad. Sci. USA 105, 5166-5171 (2008).
5. Sevignani, C., Calin, G.A., Siracusa, L.D. & Croce, C.M. Mammalian microRNAs: a small world for fne-tuning gene expression. Mamm. Genome 17, 189-202 (2006).
6. Esquela-Kerscher, A. & Slack, F.J. Oncomirs-microRNAs with a role in cancer. Nat. Rev. Cancer 6, 259-269 (2006).
7. Nicoloso, M.S., Spizzo, R., Shimizu, M., Rossi, S. & Calin, G.A. MicroRNAs-the micro steering wheel of tumour metastases. Nat. Rev. Cancer 9, 293-302 (2009).
8. Costinean, S. et al. Pre-B cell proliferation and lymphoblastic leukemia/high-grade lymphoma in E(mu)-miR155 transgenic mice. Proc. Natl. Acad. Sci. USA 103, 7024-7029 (2006).
9. Klein, U. et al. The DLEU2/miR-15a/16-1 cluster controls B cell proliferation and its deletion leads to chronic lymphocytic leukemia. Cancer Cell 17, 28-40 (2010).
10. Spizzo, R., Rushworth, D., Guerrero, M. & Calin, G.A. RNA inhibition, microRNAs, and new therapeutic agents for cancer treatment. Clin. Lymphoma Myeloma 9(suppl. 3), S313-S318 (2009).
11. Wang, H.W. et al. Structural insights into RNA processing by the human RISC-loading complex. Nat. Struct. Mol. Biol. 16, 1148-1153 (2009).
12. Blount, K.F. & Breaker, R.R. Riboswitches as antibacterial drug targets. Nat. Biotechnol. 24, 1558-1564 (2006).
13. Thomas, J.R. & Hergenrother, P.J. Targeting RNA with small molecules. Chem. Rev. 108, 1171-1224 (2008).
14. Place, R.F., Li, L.C., Pookot, D., Noonan, E.J. & Dahiya, R. MicroRNA-373 induces expression of genes with complementary promoter sequences. Proc. Natl. Acad. Sci. USA 105, 1608-1613 (2008).
15. Eiring, A.M. et al. miR-328 functions as an RNA decoy to modulate hnRNP E2 regulation of mRNA translation in leukemic blasts. Cell 140, 652-665 (2010).
16. Gumireddy, K., Young, D.D., Xiong, X., Hogenesch, J.B., Huang, Q. & Deiters, A. Small-molecule inhibitors of microrna miR-21 function. Angew. Chem. Int. Ed. Engl. 47, 7482-7484 (2008).
17. Shan, G. et al. A small molecule enhances RNA interference and promotes microRNA processing. Nat. Biotechnol. 26, 933-940 (2008).
18. Lu, J. et al. MicroRNA-mediated control of cell fate in megakaryocyte-erythrocyte progenitors. Dev. Cell 14, 843-853 (2008).
19. Detering, C. & Varani, G. Validation of automated docking programs for docking and database screening against RNA drug targets. J. Med. Chem. 47, 4188-4201 (2004).
20. Foloppe, N., Matassova, N. & Aboul-Ela, F. Towards the discovery of drug-like RNA ligands? Drug Discov. Today 11, 1019-1027 (2006).
21. Moitessier, N., Westhof, E. & Hanessian, S. Docking of aminoglycosides to hydrated and fexible RNA. J. Med. Chem. 49, 1023-1033 (2006).
22. Osterberg, F., Morris, G.M., Sanner, M.F., Olson, A.J. & Goodsell, D.S. Automated docking to multiple target structures: incorporation of protein mobility and structural water heterogeneity in AutoDock. Proteins 46, 34-40 (2002).
23. Lind, K.E., Du, Z., Fujinaga, K., Peterlin, B.M. & James, T.L. Structure-based computational database screening, in vitro assay, and NMR assessment of compounds that target TAR RNA. Chem. Biol. 9, 185-193 (2002).
24. Shapiro, B.A., Yingling, Y.G., Kasprzak, W. & Bindewald, E. Bridging the gap in RNA structure prediction. Curr. Opin. Struct. Biol. 17, 157-165 (2007).
25. Parisien, M. & Major, F. The MC-Fold and MC-Sym pipeline infers RNA structure from sequence data. Nature 452, 51-55 (2008).
26. Das, R. & Baker, D. Automated de novo prediction of native-like RNA tertiary structures. Proc. Natl. Acad. Sci. USA 104, 14664-14669 (2007).
27. Pfefer, P. & Gohlke, H. DrugScoreRNA-knowledge-based scoring function to predict RNA-ligand interactions. J. Chem. Inf. Model. 47, 1868-1876 (2007).
28. Calin, G.A. & Croce, C.M. Chronic lymphocytic leukemia: interplay between noncoding RNAs and protein-coding genes. Blood 114, 4761-4770 (2009).
29. Voorhoeve, P.M. et al. A genetic screen implicates miRNA-372 and miRNA-373 as oncogenes in testicular germ cell tumors. Cell 124, 1169-1181 (2006).