Characterizing antiprion compounds based on their binding properties to prion proteins: Implications as medical chaperones

Protein Science

Volumn 22, Issue 1, 2013, Pages 22-34

  Kamatari, Yuji O ^a   Hayano, Yosuke ^a   Yamaguchi, Kei Ichi ^a   Hosokawa Muto, Junji ^a,c   Kuwata, Kazuo ^a,b  

^a GIFU UNIVERSITY   (Japan)

^b JAPAN SCIENCE AND TECHNOLOGY AGENCY   (Japan)

^c NATIONAL RESEARCH INSTITUTE OF POLICE SCIENCE   (Japan)

Author keywords

Action mechanism; Anti prion compounds; Medical chaperones; Prion protein

Indexed keywords

CHAPERONE; CONGO RED; CP 60; D PEN; EDARAVONE DERIVATIVE 13; EPIGALLOCATECHIN GALLATE; GJP14; GJP49; INDOLE 3 GLYOXYLAMIDE DERIVATIVE 10; INDOLE 3 GLYOXYLAMIDE DERIVATIVE 12; INDOLE 3 GLYOXYLAMIDE DERIVATIVE 13; INDOLE 3 GLYOXYLAMIDE DERIVATIVE 2; MEPACRINE; PENTOSAN POLYSULFATE; PRION PROTEIN; PRION PROTEIN ANTIBODY; UNCLASSIFIED DRUG;

ARTICLE; BINDING AFFINITY; BINDING SITE; DRUG MECHANISM; DRUG RECEPTOR BINDING; DRUG STRUCTURE; MOUSE; NONHUMAN; NUCLEAR MAGNETIC RESONANCE SPECTROSCOPY; PRIORITY JOURNAL; PROTEIN BINDING; PROTEIN CONFORMATION; SURFACE PLASMON RESONANCE;

AMIDES; AMINOPYRIDINES; ANIMALS; BINDING SITES; CARBAZOLES; CATECHIN; CONGO RED; MAGNETIC RESONANCE SPECTROSCOPY; MICE; MOLECULAR CHAPERONES; MOLECULAR STRUCTURE; NITRILES; PENTOSAN SULFURIC POLYESTER; PIPERIDINES; PRIONS; PROTEIN CONFORMATION; QUINACRINE; SURFACE PLASMON RESONANCE;

EID: 84873045835     PISSN: 09618368     EISSN: 1469896X     Source Type: Journal    
DOI: 10.1002/pro.2180     Document Type: Article

References (49)

1. Prusiner SB (1998) Prions. Proc Natl Acad Sci USA 95: 13363-13383.
2. Chesebro B (1999) Prion protein and the transmissible spongiform encephalopathy diseases. Neuron 24: 503-506.
3. Prusiner SB (1982) Novel proteinaceous infectious particles cause scrapie. Science 216:136-144.
4. O'Riordan JF, Goldstick TK, Ditzel J, Ernest JT (1983) Characterization of oxygen-hemoglobin equilibrium curves using nonlinear regression of the Hill equation: parameter values for normal human adults. Adv Exp Med Biol 159:435-444.
5. Harrison PM, Bamborough P, Daggett V, Prusiner SB, Cohen FE (1997) The prion folding problem. Curr Opin Struct Biol 7:53-59.
6. Kuwata K, Nishida N, Matsumoto T, Kamatari YO, Hosokawa-Muto J, Kodama K, Nakamura HK, Kimura K, Kawasaki M, Takakura Y, Shirabe S, Takata J, Kataoka Y, Katamine S (2007) Hot spots in prion protein for pathogenic conversion. Proc Natl Acad Sci USA 104:11921-11926.
7. Hosokawa-Muto J, Kamatari YO, Nakamura HK, Kuwata K (2009) Variety of antiprion compounds discovered through an in silico screen based on cellularform prion protein structure: Correlation between antiprion activity and binding affinity. Antimicrob Agents Chemother 53:765-771.
8. Kimura T, Hosokawa-Muto J, Kamatari YO, Kuwata K (2011) Synthesis of GN8 derivatives and evaluation of their antiprion activity in TSE-infected cells. Bioorg Med Chem Lett 21:1502-1507.
9. Kimura T, Hosokawa-Muto J, Asami K, Murai T, Kuwata K (2011) Synthesis of 9-substituted 2,3,4,9-tetrahydro- 1H-carbazole derivatives and evaluation of their anti-prion activity in TSE-infected cells. Eur J Med Chem 46:5675-5679.
10. Doh-Ura K, Iwaki T, Caughey B (2000) Lysosomotropic agents and cysteine protease inhibitors inhibit scrapieassociated prion protein accumulation. J Virol 74: 4894-4897.
11. Perrier V, Wallace AC, Kaneko K, Safar J, Prusiner SB, Cohen FE (2000) Mimicking dominant negative inhibition of prion replication through structure-based drug design. Proc Natl Acad Sci USA 97:6073-6078.
12. Kocisko DA, Baron GS, Rubenstein R, Chen J, Kuizon S, Caughey B (2003) New inhibitors of scrapie-associated prion protein formation in a library of 2000 drugs and natural products. J Virol 77:10288-10294.
13. Korth C, May BC, Cohen FE, Prusiner SB (2001) Acridine and phenothiazine derivatives as pharmacotherapeutics for prion disease. Proc Natl Acad Sci USA 98: 9836-9841.
14. Caughey B, Ernst D, Race RE (1993) Congo red inhibition of scrapie agent replication. J Virol 67:6270-6272.
15. Rambold AS, Miesbauer M, Olschewski D, Seidel R, Riemer C, Smale L, Brumm L, Levy M, Gazit E, Oesterhelt D, Baier M, Becker CF, Engelhard M, Winklhofer KF, Tatzelt J (2008) Green tea extracts interfere with the stress-protective activity of PrP and the formation of PrP. J Neurochem 107:218-229.
16. Doh-ura K, Ishikawa K, Murakami-Kubo I, Sasaki K, Mohri S, Race R, Iwaki T (2004) Treatment of transmissible spongiform encephalopathy by intraventricular drug infusion in animal models. J Virol 78: 4999-5006.
17. Tsuboi Y, Doh-Ura K, Yamada T (2009) Continuous intraventricular infusion of pentosan polysulfate: clinical trial against prion diseases. Neuropathology 29:632-636.
18. Kimata A, Nakagawa H, Ohyama R, Fukuuchi T, Ohta S, Doh-ura K, Suzuki T, Miyata N (2007) New series of antiprion compounds: pyrazolone derivatives have the potent activity of inhibiting protease-resistant prion protein accumulation. J Med Chem 50:5053-5056.
19. Sigurdsson EM, Brown DR, Alim MA, Scholtzova H, Carp R, Meeker HC, Prelli F, Frangione B, Wisniewski T (2003) Copper chelation delays the onset of prion disease. J Biol Chem 278:46199-46202.
20. Thompson MJ, Borsenberger V, Louth JC, Judd KE, Chen B (2009) Design, synthesis, and structure-activity relationship of indole-3-glyoxylamide libraries possessing highly potent activity in a cell line model of prion disease. J Med Chem 52:7503-7511.
21. Kuwata K, Kamatari YO, Akasaka K, James TL (2004) Slow conformational dynamics in the hamster prion protein. Biochemistry 43:4439-4446.
22. Nakajima M, Yamada T, Kusuhara T, Furukawa H, Takahashi M, Yamauchi A, Kataoka Y (2004) Results of quinacrine administration to patients with Creutzfeldt- Jakob disease. Dement Geriatr Cogn Disord 17: 158-163.
23. Milhavet O, McMahon HE, Rachidi W, Nishida N, Katamine S, Mange A, Arlotto M, Casanova D, Riondel J, Favier A, Lehmann S (2000) Prion infection impairs the cellular response to oxidative stress. Proc Natl Acad Sci USA 97:13937-13942.
24. Vogtherr M, Grimme S, Elshorst B, Jacobs DM, Fiebig K, Griesinger C, Zahn R (2003) Antimalarial drug quinacrine binds to C-terminal helix of cellular prion protein. J Med Chem 46:3563-3564.
25. Telling GC, Scott M, Mastrianni J, Gabizon R, Torchia M, Cohen FE, DeArmond SJ, Prusiner SB (1995) Prion propagation in mice expressing human and chimeric PrP transgenes implicates the interaction of cellular PrP with another protein. Cell 83:79-90.
26. Masel J, Genoud N, Aguzzi A (2005) Efficient inhibition of prion replication by PrP-Fc(2) suggests that the prion is a PrP(Sc) oligomer. J Mol Biol 345:1243-1251.
27. Wille H, Zhang GF, Baldwin MA, Cohen FE, Prusiner SB (1996) Separation of scrapie prion infectivity from PrP amyloid polymers. J Mol Biol 259:608-621.
28. Pham N, Yin S, Yu S, Wong P, Kang SC, Li C, Sy MS (2008) Normal cellular prion protein with a methionine at position 129 has a more exposed helix 1 and is more prone to aggregate. Biochem Biophys Res Commun 368:875-881.
29. Lamberto GR, Binolfi A, Orcellet ML, Bertoncini CW, Zweckstetter M, Griesinger C, Fernandez CO (2009) Structural and mechanistic basis behind the inhibitory interaction of PcTS on alpha-synuclein amyloid fibril formation. Proc Natl Acad Sci USA 106:21057-21062.
30. Nicoll AJ, Trevitt CR, Tattum MH, Risse E, Quarterman E, Ibarra AA, Wright C, Jackson GS, Sessions RB, Farrow M, Waltho JP, Clarke AR, Collinge J (2010) Pharmacological chaperone for the structured domain of human prion protein. Proc Natl Acad Sci USA 107: 17610-17615.
31. Ansari A, Berendzen J, Bowne SF, Frauenfelder H, Iben IE, Sauke TB, Shyamsunder E, Young RD (1985) Protein states and proteinquakes. Proc Natl Acad Sci USA 82:5000-5004.
32. Itoh K, Sasai M (2004) Dynamical transition and proteinquake in photoactive yellow protein. Proc Natl Acad Sci USA 101:14736-14741.
33. Cohen FE, Prusiner SB (1998) Pathologic conformations of prion proteins. Annu Rev Biochem 67:793-819.
34. Cohen FE, Pan KM, Huang Z, Baldwin M, Fletterick RJ, Prusiner SB (1994) Structural clues to prion replication. Science 264:530-531.
35. Cohen FE, Prusiner SB, Structural studies of prion proteins. In: Prusiner SB, Ed. (1999) Prion biology and diseases. New York: Cold Spring Harbor, pp191-228.
36. Yamamoto N, Kuwata K (2009) Regulating the conformation of prion protein through ligand binding. J Phys Chem B 113:12853-12856.
37. Telling GC, Parchi P, DeArmond SJ, Cortelli P, Montagna P, Gabizon R, Mastrianni J, Lugaresi E, Gambetti P, Prusiner SB (1996) Evidence for the conformation of the pathologic isoform of the prion protein enciphering and propagating prion diversity. Science 274: 2079-2082.
38. Safar J, Wille H, Itri V, Groth D, Serban H, Torchia M, Cohen FE, Prusiner SB (1998) Eight prion strains have PrP(Sc) molecules with different conformations. Nat Med 4:1157-1165.
39. Kawasaki Y, Kawagoe K, Chen CJ, Teruya K, Sakasegawa Y, Doh-ura K (2007) Orally administered amyloidophilic compound is effective in prolonging the incubation periods of animals cerebrally infected with prion diseases in a prion strain-dependent manner. J Virol 81:12889-12898.
40. Li J, Browning S, Mahal SP, Oelschlegel AM, Weissmann C (2010) Darwinian evolution of prions in cell culture. Science 327:869-872.
41. Sawkar AR, Cheng WC, Beutler E, Wong CH, Balch WE, Kelly JW (2002) Chemical chaperones increase the cellular activity of N370S beta -glucosidase: a therapeutic strategy for Gaucher disease. Proc Natl Acad Sci USA 99:15428-15433.
42. Kanu N, Imokawa Y, Drechsel DN, Williamson RA, Birkett CR, Bostock CJ, Brockes JP (2002) Transfer of scrapie prion infectivity by cell contact in culture. Curr Biol 12:523-530.
43. Montagnier L (2010) 25 years after HIV discovery: prospects for cure and vaccine. Virology 397:248-254.
44. Shafer RW, Vuitton DA (1999) Highly active antiretroviral therapy (HAART) for the treatment of infection with human immunodeficiency virus type 1. Biomed Pharmacother 53:73-86.
45. Roberts BE, Duennwald ML, Wang H, Chung C, Lopreiato NP, Sweeny EA, Knight MN, Shorter J (2009) A synergistic small-molecule combination directly eradicates diverse prion strain structures. Nat Chem Biol 5:936-946.
46. Hornemann S, Korth C, Oesch B, Riek R, Wider G, Wuthrich K, Glockshuber R (1997) Recombinant fulllength murine prion protein, mPrP(23-231): purification and spectroscopic characterization. FEBS Lett 413: 277-281.
47. Goddard TD, Kneller DG (2001) SPARKY, version 3. San Francisco: University of California.
48. Riek R, Hornemann S, Wider G, Billeter M, Glockshuber R, Wuthrich K (1996) NMR structure of the mouse prion protein domain PrP(121-231). Nature 382: 180-182.
49. Nishida N, Harris DA, Vilette D, Laude H, Frobert Y, Grassi J, Casanova D, Milhavet O, Lehmann S (2000) Successful transmission of three mouse-adapted scrapie strains to murine neuroblastoma cell lines overexpressing wild-type mouse prion protein. J Virol 74:320-325.