Rationally Designed Small Molecules That Target Both the DNA and RNA Causing Myotonic Dystrophy Type 1

Journal of the American Chemical Society

Volumn 137, Issue 44, 2015, Pages 14180-14189

  Nguyen, Lien ^a   Luu, Long M ^a   Peng, Shaohong ^b   Serrano, Julio F ^a   Chan, H Y Edwin ^b   Zimmerman, Steven C ^a  

^a UNIVERSITY OF ILLINOIS AT URBANA CHAMPAIGN   (United States)

^b CHINESE UNIVERSITY OF HONG KONG   (Hong Kong)

Author keywords

[No Author keywords available]

Indexed keywords

BINS; BIOCHEMISTRY; MOLECULES; PROTEINS; RNA; TRANSCRIPTION;

ABNORMAL EXPANSION; COMPLEX DISEASE; MULTI-TARGETS; MYOTONIC DYSTROPHY; POTENT COMPOUNDS; PROTEIN BINDING; SINGLE-AGENT; SMALL MOLECULES;

NUCLEIC ACIDS;

AGENTS AFFECTING NUCLEIC ACID METABOLISM; DNA; LIGAND; RIBONUCLEASE; RNA; MOLECULAR LIBRARY;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DRUG DESIGN; DRUG TARGETING; FRUIT FLY MODEL; HUMAN; HUMAN CELL; IN VITRO STUDY; MYOTONIC DYSTROPHY; NERVE DEGENERATION; NONHUMAN; PHENOTYPE; PROTEIN BINDING; RNA CLEAVAGE; TRINUCLEOTIDE REPEAT; ANIMAL; BIOSYNTHESIS; CHEMISTRY; DISEASE MODEL; DROSOPHILA; DRUG EFFECTS; GENETICS; HELA CELL LINE; METABOLISM; MOLECULAR LIBRARY; PATHOLOGY; PHARMACOLOGY; SYNTHESIS;

ANIMALS; DISEASE MODELS, ANIMAL; DNA; DROSOPHILA; DRUG DESIGN; HELA CELLS; HUMANS; MYOTONIC DYSTROPHY; RNA; SMALL MOLECULE LIBRARIES; TRINUCLEOTIDE REPEAT EXPANSION;

EID: 84947465842     PISSN: 00027863     EISSN: 15205126     Source Type: Journal    
DOI: 10.1021/jacs.5b09266     Document Type: Article

References (42)

1. Kunnumakkara, A. B.; Anand, P.; Aggarwal, B. B. Cancer Lett. 2008, 269, 199-225 10.1016/j.canlet.2008.03.009
2. Imming, P.; Sinning, C.; Meyer, A. Nat. Rev. Drug Discovery 2006, 5, 821-834 10.1038/nrd2132
3. Anighoro, A.; Bajorath, J.; Rastelli, G. J. Med. Chem. 2014, 57, 7874-7887 10.1021/jm5006463
4. Morphy, R.; Rankovic, Z. J. Med. Chem. 2005, 48, 6523-6543 10.1021/jm058225d
5. Meunier, B. Acc. Chem. Res. 2008, 41, 69-77 10.1021/ar7000843
6. Gatchel, J. R.; Zoghbi, H. Y. Nat. Rev. Genet. 2005, 6, 743-755 10.1038/nrg1691
7. Wheeler, T. M.; Sobczak, K.; Lueck, J. D.; Osborne, R. J.; Lin, X.; Dirksen, R. T.; Thornton, C. A. Science 2009, 325, 336-339 10.1126/science.1173110
8. Gareiss, P. C.; Sobczak, K.; McNaughton, B. R.; Palde, P. B.; Thornton, C. A.; Miller, B. L. J. Am. Chem. Soc. 2008, 130, 16254-16261 10.1021/ja804398y
9. Warf, M. B.; Nakamori, M.; Matthys, C. M.; Thornton, C. A.; Berglund, J. A. Proc. Natl. Acad. Sci. U. S. A. 2009, 106, 18551-18556 10.1073/pnas.0903234106
10. Parkesh, R.; Childs-Disney, J. L.; Nakamori, M.; Kumar, A.; Wang, E.; Wang, T.; Hoskins, J.; Tran, T.; Housman, D.; Thornton, C. A.; Disney, M. D. J. Am. Chem. Soc. 2012, 134, 4731-4742 10.1021/ja210088v
11. Arambula, J. F.; Ramisetty, S. R.; Baranger, A. M.; Zimmerman, S. C. Proc. Natl. Acad. Sci. U. S. A. 2009, 106, 16068-16073 10.1073/pnas.0901824106
12. Wong, C.-H.; Nguyen, L.; Peh, J.; Luu, L. M.; Sanchez, J. S.; Richardson, S. L.; Tuccinardi, T.; Tsoi, H.; Chan, W. Y.; Chan, H. Y. E.; Baranger, A. M.; Hergenrother, P. J.; Zimmerman, S. C. J. Am. Chem. Soc. 2014, 136, 6355-6361 10.1021/ja5012146
13. García-López, A.; Llamusí, B.; Orzáez, M.; Pérez-Payá, E.; Artero, R. D. Proc. Natl. Acad. Sci. U. S. A. 2011, 108, 11866-11871 10.1073/pnas.1018213108
14. Jahromi, A. H.; Honda, M.; Zimmerman, S. C.; Spies, M. Nucleic Acids Res. 2013, 41, 6687-6697 10.1093/nar/gkt330
15. Cho, D. H.; Thienes, C. P.; Mahoney, S. E.; Analau, E.; Filippova, G. N.; Tapscott, S. J. Mol. Cell 2005, 20, 483-489 10.1016/j.molcel.2005.09.002
16. Moseley, M. L.; Zu, T.; Ikeda, Y.; Gao, W.; Mosemiller, A. K.; Daughters, R. S.; Chen, G.; Weatherspoon, M. R.; Clark, H. B.; Ebner, T. J.; Day, J. W.; Ranum, L. P. W. Nat. Genet. 2006, 38, 758-769 10.1038/ng1827
17. Zu, T.; Gibbens, B.; Doty, N. S.; Gomes-Pereira, M.; Huguet, A.; Stone, M. D.; Margolis, J.; Peterson, M.; Markowski, T. W.; Ingram, M. A. C.; Nan, Z.; Forster, C.; Low, W. C.; Schoser, B.; Somia, N. V.; Clark, H. B.; Schmechel, S.; Bitterman, P. B.; Gourdon, G.; Swanson, M. S.; Moseley, M.; Ranum, L. P. W. Proc. Natl. Acad. Sci. U. S. A. 2011, 108, 260-265 10.1073/pnas.1013343108
18. Wojciechowska, M.; Olejniczak, M.; Galka-Marciniak, P.; Jazurek, M.; Krzyzosiak, W. J. Nucleic Acids Res. 2014, 42, 11849-11864 10.1093/nar/gku794
19. Kalsotra, A.; Singh, R. K.; Gurha, P.; Ward, A. J.; Creighton, C. J.; Cooper, T. A. Cell Rep. 2014, 6, 336-345 10.1016/j.celrep.2013.12.025
20. Laurent, F. X.; Sureau, A.; Klein, A. F.; Trouslard, F.; Gasnier, E.; Furling, D.; Marie, J. Nucleic Acids Res. 2012, 40, 3159-3171 10.1093/nar/gkr1228
21. Pettersson, O. J.; Aagaard, L.; Andrejeva, D.; Thomsen, R.; Jensen, T. G.; Damgaard, C. K. Nucleic Acids Res. 2014, 42, 7186-7200 10.1093/nar/gku352
22. Lee, J. E.; Bennett, C. F.; Cooper, T. A. Proc. Natl. Acad. Sci. U. S. A. 2012, 109, 4221-4226 10.1073/pnas.1117019109
23. Wheeler, T. M.; Leger, A. J.; Pandey, S. K.; MacLeod, A. R.; Nakamori, M.; Cheng, S. H.; Wentworth, B. M.; Bennett, C. F.; Thornton, C. A. Nature 2012, 488, 111-115 10.1038/nature11362
24. Guan, L.; Disney, M. D. Angew. Chem., Int. Ed. 2013, 52, 1462-1465 10.1002/anie.201206888
25. Coonrod, L. A.; Nakamori, M.; Wang, W.; Carrell, S.; Hilton, C. L.; Bodner, M. J.; Siboni, R. B.; Docter, A. G.; Haley, M. M.; Thornton, C. A.; Berglund, J. A. ACS Chem. Biol. 2013, 8, 2528-2537 10.1021/cb400431f
26. Wong, C.-H.; Richardson, S. L.; Ho, Y.-J.; Lucas, A. M. H.; Tuccinardi, T.; Baranger, A. M.; Zimmerman, S. C. ChemBioChem 2012, 13, 2505-2509 10.1002/cbic.201200602
27. Jahromi, A. H.; Nguyen, L.; Fu, Y.; Miller, K. A.; Baranger, A. M.; Zimmerman, S. C. ACS Chem. Biol. 2013, 8, 1037-1043 10.1021/cb400046u
28. Palmer, A. J.; Wallace, H. M. Amino Acids 2010, 38, 415-422 10.1007/s00726-009-0400-2
29. Trawick, B. N.; Daniher, A. T.; Bashkin, J. K. Chem. Rev. 1998, 98, 939-960 10.1021/cr960422k
30. Lönnberg, H. Org. Biomol. Chem. 2011, 9, 1687-1703 10.1039/c0ob00486c
31. Breslow, R.; Huang, D. L.; Anslyn, E. Proc. Natl. Acad. Sci. U. S. A. 1989, 86, 1746-1750 10.1073/pnas.86.6.1746
32. Miller, J. W.; Urbinati, C. R.; Teng-Umnuay, P.; Stenberg, M. G.; Byrne, B. J.; Thornton, C. A.; Swanson, M. S. EMBO J. 2000, 19, 4439-4448 10.1093/emboj/19.17.4439
33. Wong, C. H.; Fu, Y.; Ramisetty, S. R.; Baranger, A. M.; Zimmerman, S. C. Nucleic Acids Res. 2011, 39, 8881-8890 10.1093/nar/gkr415
34. Savkur, R. S.; Philips, A. V.; Cooper, T. A. Nat. Genet. 2001, 29, 40-47 10.1038/ng704
35. Lanson, N. A.; Maltare, A.; King, H.; Smith, R.; Kim, J. H.; Taylor, J. P.; Lloyd, T. E.; Pandey, U. B. Hum. Mol. Genet. 2011, 20, 2510-2523 10.1093/hmg/ddr150
36. Nichols, C. D.; Becnel, J.; Pandey, U. B. J. Visualized Exp. 2012, 61, e3795 10.3791/3795
37. Batlevi, Y.; Martin, D. N.; Pandey, U. B.; Simon, C. R.; Powers, C. M.; Taylor, J. P.; Baehrecke, E. H. Proc. Natl. Acad. Sci. U. S. A. 2010, 107, 742-747 10.1073/pnas.0907967107
38. Schmittgen, T. D.; Livak, K. J. Nat. Protoc. 2008, 3, 1101-1108 10.1038/nprot.2008.73
39. Garcia-Lopez, A.; Monferrer, L.; Garcia-Alcover, I. PLoS One 2008, 3, e1595 10.1371/journal.pone.0001595
40. Garcia-Alcover, I.; Colonques-Bellmunt, J.; Garijo, R.; Tormo, J. R.; Artero, R.; álvarez-Abril, M. C.; López Castel, A.; Pérez-Alonso, M. Dis. Models & Mech. 2014, 7, 1297-1306 10.1242/dmm.016592
41. Freeman, M. Cell 1996, 87, 651-660 10.1016/S0092-8674(00)81385-9
42. Brand, A. H.; Perrimon, N. Development 1993, 118, 401-415