Small-molecule modulation of cellular chaperones to treat protein misfolding disorders

Current Opinion in Drug Discovery and Development

Volumn 12, Issue 5, 2009, Pages 666-681

  Sloan, Lisa A ^a   Fillmore, Martin C ^a   Churcher, Ian ^a  

^a ClaxoSmlthKllne Medicines Research Centre   (United Kingdom)

Author keywords

Chaperone; Heat shock; HSF 1; Hsp70; Potentiator; Protein misfolding

Indexed keywords

AEG 3482; ARIMOCLOMOL; BENZYLOXYCARBONYLLEUCYLLEUCYLLEUCINAL; BORTEZOMIB; BRX 220; CARBENOXOLONE; CELASTROL; CHAPERONE; CURCUMIN; ELESCLOMOL; ESTER DERIVATIVE; FERULIC ACID ETHYL ESTER; GELDANAMYCIN; GLYCYRRHIZIC ACID; HEAT SHOCK PROTEIN; HYDROXYLAMINE; INDOMETACIN; KETONE DERIVATIVE; KNK 437; LACTACYSTIN; N ALPHA TOSYL LYSINE CHLOROMETHYL KETONE; NOVOBIOCIN; O (2 HYDROXY 3 PIPERIDINOPROPYL)NICOTINIC AMIDOXIME; PIOGLITAZONE; PROTEIN INHIBITOR; PUROMYCIN; QUERCETIN; RESVERATROL; RILUZOLE; SALICYLIC ACID; TEPRENONE; TOSYLLYSYL CHLOROMETHYL KETONE; UNCLASSIFIED DRUG; UNINDEXED DRUG;

ALPHA 1 ANTITRYPSIN DEFICIENCY; ALZHEIMER DISEASE; AMYLOID NEUROPATHY; AMYLOIDOSIS; AMYOTROPHIC LATERAL SCLEROSIS; ATROPHY; CLINICAL TRIAL; CREUTZFELDT JAKOB DISEASE; CYSTIC FIBROSIS; CYTOSOL; DENTATORUBAL PALLIDOLUYSIAL ATROPHY; DRUG POTENCY; DRUG STRUCTURE; FABRY DISEASE; FAMILIAL AMYLOID POLYNEUROPATHY; FAMILIAL AMYLOIDOSIS; FAMILIAL MEDITERRANEAN FEVER; FATAL FAMILIAL INSOMNIA; FRAGILE X SYNDROME; GANGLIOSIDOSIS; GAUCHER DISEASE; GENETIC REGULATION; GLOBOID CELL LEUKODYSTROPHY; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; HUNTINGTON CHOREA; HYPOGONADOTROPIC HYPOGONADISM; KENNEDY DISEASE; MACHADO JOSEPH DISEASE; MEDULLARY CARCINOMA; MELANOMA; NEPHROGENIC DIABETES INSIPIDUS; NIEMANN PICK DISEASE; NON INSULIN DEPENDENT DIABETES MELLITUS; NONHUMAN; OBESITY; PARKINSON DISEASE; PATHOGENESIS; PROTEIN DEFECT; PROTEIN EXPRESSION; PROTEIN FOLDING; PROTEIN FUNCTION; PROTEIN PROCESSING; PROTEIN SYNTHESIS INHIBITION; REVIEW; SANDHOFF DISEASE; SICKLE CELL ANEMIA; STOMACH ULCER; TAUOPATHY; TAY SACHS DISEASE; UPREGULATION; VASCULAR AMYLOIDOSIS;

ANIMALS; DRUG DESIGN; GENE THERAPY; HSP70 HEAT-SHOCK PROTEINS; HSP90 HEAT-SHOCK PROTEINS; HUMANS; HYDROXYLAMINES; MOLECULAR CHAPERONES; MOLECULAR STRUCTURE; PROTEIN CONFORMATION; PROTEIN FOLDING; PROTEIN PROCESSING, POST-TRANSLATIONAL; PROTEOSTASIS DEFICIENCIES; STRUCTURE-ACTIVITY RELATIONSHIP; UP-REGULATION;

EID: 70149093873     PISSN: 13676733     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (166)

1. Hoff H, Zhang H, Sell C: Protein degradation via the proteosome. Methods Mol Biol (2004) 285:79-92.
2. Suty L: The ubiquitin-proteosome proteolytic system. From classical biochemistry to human diseases. Recent advances in human biology Vol9. Cell Biochem Funct (2004) 22(3):205.
3. Scarlatti F, Maffei R, Beau I, Ghidoni R, Codogno P; Non-canonical autophagy: An exception or an underestimated form of autophagy? Autophagy (2008) 4(8):1083-1085.
4. Dengjel J, Kristensen AR, Andersen JS; Ordered bulk degradation via autophagy. Autophagy (2008) 4(8): 1057-1059.
5. Todde V, Veenhuis M, van der Klei IJ; Autophagy: Principles and significance in health and disease. Biochim Biophys Acta (2009) 1792(1):3-13.
6. Chang H, Tang Y, Hayer-Hartl M, Haiti FU: Snapshot: Molecular chaperones. Part I. Cell (2007) 128(1):212.
7. Tang YC, Chang HC, Hayer-Hartl M, Hartl FU: Snapshot: Molecular chaperones, Part II. Cell (2007) 128(2);412.
8. Ran R, Lu A, Xu H, Tang Y, Sharp FR: Heat-shock protein regulation of protein folding, protein degradation, protein function and apoptosis, In: Handbook of Neurochemistry and Molecular Neurobiology Acute Ischemic Injury and Repair in the Nervous System. Lajtha A, Chan PH (Eds), Springer, USA (2007):89-107.
9. 9. Zhao L, Ackerman SL: Endoplasmic reticulum stress in health and disease. Curr Opin Cell Biol (2006) 18(4):444-452. (Pubitemid 44026518)
10. 10. Kaufman RJ: Orchestrating the unfolded protein response in health and disease. J Clin Invest (2002) 110(10): 1389-1398. (Pubitemid 35396907)
11. Forman MS, Lee VMY, Trojanowski JQ: 'Unfolding' pathways in neurodegenerative disease. Trends Neurosci (2003) 26(8): 407-410.
12. 12. Czarnecka AM, Campanella C, Zummo G, Cappello F: Mitochondrial chaperones in cancer: From molecular biology to clinical diagnostics. Cancer Biol Ther (2006) 5(7): 714-720. (Pubitemid 44775122)
13. Horváth I, Multhoff G, Sonnleitner A, Vigh L; Membrane-associated stress proteins: More than simply chaperones. Biochim Biophys Acta (2008) 1778(7-8): 1653-1664.
14. Macario AJL, De Macario EC; Chaperonopathies by defect, excess or mistake. Ann NY Acad Sci (2007) 1113:178-191.
15. Macario AJL, Grippo TM, Conway De Macario E; Genetic disorders involving molecular-chaperone genes: A perspective. Genet Med (2005) 7(1);3-12.
16. 16. Hayashi M, Imanaka-Yoshida K, Yoshida T, Wood M, Fearns C, Tatake RJ, Lee JD; A crucial role of mitochondrial Hsp40 in preventing dilated cardiomyopathy. Nat Med (2006) 12(1); 128-132. (Pubitemid 43050087)
17. 17. Stock AD, Spallone PA, Dennis TR, Netski D, Morris CA, Mervis CB, Hobart HH; Heat shock protein 27 gene: Chromosomal and molecular location and relationship to Williams syndrome. Am J Med Genet A (2003) 120A(3); 320-325. (Pubitemid 37063765)
18. 18. Harrison PJ, Procter AW, Exworthy T, Roberts GW, Najlerahim A, Abarton AJL, Pearson RCA; Heat shock protein (Hsx70) mRNA expression in human brain: Effect of neurodegenerative disease and agonal state. Neuropathol Appl Neurobiol (1993) 19(1):10-21. (Pubitemid 23092977)
19. Wakutani Y, Urakami K, Shimomura T, Takahashi K: Heat shock protein 70 mRNA levels in mononuclear blood cells from patients with dementia of the Alzheimer type. Dementia (1995) 6(6):301-305.
20. Wu C: Heat shock transcription factors: Structure and regulation. Annu Rev Cell Dev Biol (1995) 11:441-469.
21. 21. Pirkkala L, Nykanen P, Sistonen L: Roles of the heat shock transcription factors in regulation of the heat shock response and beyond. FASEB J (2001) 15(7):1118-1131. (Pubitemid 32436297)
22. 22. Amin J, Fernandez M, Ananthan J, Lis JT, Voellmy R: Cooperative binding of heat shock transcription factor to the Hsp70 promoter in vivo and i. J Biol Chem (1994) 269(7): 4804-4811. (Pubitemid 24239453)
23. 23. Whitesell L, Bagatell R, Falsey R: The stress response: Implications for the clinical development of Hsp90 inhibitors. Curr Cancer Drug Targets (2003) 3(5):349-358. (Pubitemid 37128321)
24. Baiogh G, Horvath I, Nagy E, Hoyk Z, Benko S, Bensaude O, Vigh L: The hyperfluidization of mammalian cell membranes acts as a signal to initiate the heat shock protein response. FESS J (2005) 272(23);6077-6086.
25. Zuo J, Rungger D, Voellmy R: Multiple layers of regulation of human heat shock transcription factor 1. Mol Cell Biol (1995) 15(8):4319-4330.
26. Marber MS, Mestril R, Chi SH, Sayen MR, Yellon DM, Dillmann WH; Overexpression of the rat inducible 70-kD heat stress protein in a transgenic mouse increases the resistance of the heart to ischemic injury. J Clin Invest (1995) 95(4); 1446-1456.
27. 27. Chung J, Nguyen AK, Henstridge DC, Holmes AG, Chan MHS, Mesa JL, Lancaster GI, Southgate RJ, Bruce CR, Duffy SJ, Horvath I et al: Hsp72 protects against obesity-induced insulin resistance. Proc Natl Acad Sci USA (2008) 105(5); 1739-1744. (Pubitemid 351346584)
28. 28. Broome CS, Kayani AC, Palomero J, Dillmann WH, Mestril R, Jackson MJ, McArdle A: Effect of lifelong overexpression of Hsp70 in skeletal muscle on age-related oxidative stress and adaptation after nondamaging contractile activity. FASEB J (2006) 20(9);1549-1551. (Pubitemid 44943826)
29. Zheng Z, Kim JY, Ma H, Lee JE, Yenari MA; Anti-inflammatory effects of the 70 kDa heat shock protein in experimental stroke. J Cereb Blood Flow Metab (2008) 28(1) : 53-63.
30. 30. Muchowski PJ, Schaffar G, Sittler A, Wanker EE, Hayer-Hartl MK, Hart FU; Hsp70 and Hsp40 chaperones can inhibit self-assembly of polyglutamine proteins into amyloid-like fibrils. Proc Natl Acad Sci USA (2000) 97(14);7841-7846. (Pubitemid 30460735)
31. 508 CFTR trafficking. Am J Physiol Lung Cell Mol Physiol (2001) 281(1);L58-L68.
32. Donnelly A, Blagg BSJ; Novobiocin and additional inhibitors of the Hsp90 C-terminal nucleotide binding pocket. Curr Med Chem (2008) 15(26):2702-2717.
33. Hadden MK, Lubbers DJ, Blagg BSJ: Geldanamycin, radicicol, and chimeric inhibitors of the Hsp90 N-terminal ATP binding site. Curr Top Med Chem (2006) 6(11); 1173-1182.
34. Karapanagiotou EM, Syrigos K, Saif MW; Heat shock protein inhibitors and vaccines as new agents in cancer treatment. Expert Opin Invest Drugs (2009) 18(2) : 161-174.
35. 35. McDonald E, Workman P, Jones K: Inhibitors of the Hsp90 molecular chaperone: Attacking the master regulator in cancer. Curr Top Med Chem (2006) 6(11);1091-1107. (Pubitemid 44110922)
36. 36. Solit DB, Rosen N; Hsp90: A novel target for cancer therapy. Curr Top Med Chem (2006) 6(11):1205-1214. (Pubitemid 44120915)
37. Taldone T, Gozman A, Maharaj R, Chiosis G: Targeting Hsp90: Small-molecule inhibitors and their clinical development. Curr Opin Pharmacol (2008) 8(4):370-374.
38. 38. Herbst M, Wanker EE: Small molecule inducers of heat-shock response reduce polyQ-mediated huntingtin aggregation. A possible therapeutic strategy. Neurodegener Dis (2007) 4(2-3):254-260. (Pubitemid 47000417)
39. 39. Marcu MG, Chadli A, Bouhouche I, Catelli M, Neckers LM: The heat shock protein 90 antagonist novobiocin interacts with a previously unrecognized ATP-binding domain in the carboxyl terminus of the chaperone. J Biol Chem (2000) 275(47):37181-37186. (Pubitemid 32002137)
40. 40. Ansar S, Burlison JA, Hadden MK, Yu XM, Desino KE, Bean J, Neckers L, Audus KL, Michaelis ML, Blagg BSJ; A non-toxic Hsp90 inhibitor protects neurons from Aß-induced toxicity. Bioorg Med Chem Lett (2007) 17 (7); 1984-1990. (Pubitemid 46367655)
41. Lauricella M, Emanuele S, D'Anneo A, Calvaruso G, Vassallo B, Carlisi D, Portanova P, Vento R, Tesoriere G; JNK and AP-1 mediate apoptosis induced by bortezomib in HepG2 cells via FasL/caspase-8 and mitochondria-dependent pathways. Apoptosis (2006) 11(4):607-625.
42. 42. Kim D, Li GC; Proteasome inhibitors lactacystin and MG132 inhibit the dephosphorylation of HSF1 after heat shock and suppress thermal induction of heat shock gene expression. Biochem Biophys Res Commun (1999) 264(2):352-358. (Pubitemid 29516091)
43. 43. Kim WY, Horbinski C, Sigurdson W, Higgins D: Proteasome inhibitors suppress formation of polyglutamine-induced nuclear inclusions in cultured postmitotic neurons. J Neurochem (2004) 91(5);1044-1056. (Pubitemid 39587443)
44. Mei J, Niu C, Chen J, Ni Y: Retrograde changes of motor behavior in the rat models of Parkinson's disease induced by lactacystin. Zhongguo Weiqinxi Shenjing Waike Zazhi (2008) 13(10):466-469.
45. 45. Salehi AH, Morris SJ, Ho WC, Dickson KM, Doucet G, Milutinovic S, Durkin J, Gillard JW, Barker PA: AEG3482 is an antiapoptotic compound that inhibits Jun kinase activity and cell death through induced expression of heat shock protein 70. Chem Biol (2006) 13(2):213-223. (Pubitemid 43255593)
46. 46. Yoh T, Nakashima T, Sumida Y, Kakisaka Y, Nakajima Y, Ishikawa H, Sakamoto Y, Okanoue T, Mitsuyoshi H: Effects of glycyrrhizin on glucocorticoid signaling pathway in hepatocytes. Dig Dis Sci (2002) 47(8):1775-1781. (Pubitemid 34833208)
47. Lee YJ, Dewey WC: Induction of heat shock proteins in Chinese hamster ovary cells and development of thermotolerance by intermediate concentrations of puromycin. J Cell Physiol (1987) 132(1);1-11.
48. 2+-ATPase of sarcoplasmic reticulum by thiol-specific, heat shock inducers results in thermal denaturation at 37 degrees C. Biochemistry (1997) 36(36);11002-11011.
49. 49. Rossi A, Elia G, Santoro MG: Activation of the heat shock factor 1 by serine protease inhibitors. An effect associated with nuclear factor-kB inhibition. J Biol Chem (1998) 273(26): 16446-16452. (Pubitemid 28311427)
50. Santoro MG: Heat shock factors and the control of the stress response. Biochem Pharmacol (2000) 59(1):55-63.
51. Commandeur JNM, Vermeulen NPE; Cytotoxicity and cytoprotective activities of natural compounds. The case of curcumin. Xenobiotica (1996) 26(7):667-680.
52. Dunsmore KE, Chen PG, Wong HR: Curcumin, a medicinal herbal compound capable of inducing the heat shock response. Crit Care Med (2001) 29(11):2199-2204.
53. Kanitkar M, Bhonde RR; Curcumin treatment enhances islet recovery by induction of heat shock response proteins, Hsp70 and heme oxygenase-1, during cryopreservation. Life Sci (2008) 82(3-4): 182-189.
54. Toennesen HH, Karlsen J: Studies on curcumin and curcuminoids. VI. Kinetics of curcumin degradation in aqueous solution. Z Lebensm Unters Forsch (1985) 180(5); 402-404.
55. 55. Perluigi M, Joshi G, Sultana R, Calabrese V, De Marco C, Coccia R, Cini C, Butterfield DA; In vivo protective effects of ferulic acid ethyl ester against amyloid-p peptide 1-42-induced oxidative stress. J Neurosci Res (2006) 84(2):418-426. (Pubitemid 44126294)
56. Hamel L, Kenney M, Jayyosi Z, Ardati A, Clark K, Spada A, Zilberstein A, Perrone M, Kaplow J, Merkel L, Rojas C: Induction of heat shock protein 70 by herbimycin A and cyclopentenone prostaglandins in smooth muscle cells. Cell Stress Chaperones (2000) 5(2):121-131.
57. Lee BS, Chen J, Angelidis C, Jurivich DA, Morimoto RI; Pharmacological modulation of heat shock factor 1 by antiinflammatory drugs results in protection against stress-induced cellular damage. Proc Natl Acad Sci USA (1995) 92(16);7207-7211.
58. 58. Taniguchi Y, Ooie T, Takahashi N, Shinohara T, Nakagawa M, Yonemochi H, Hara M, Yoshimatsu H, Saikawa T: Pioglitazone but not glibenclamide improves cardiac expression of heat shock protein 72 and tolerance against ischemia/reperfusion injury in the heredity insulin-resistant rat. Diabetes (2006) 55(8);2371-2378. (Pubitemid 44743637)
59. 59. Westerheide SD, Bosman JD, Mbadugha BNA, Kawahara TLA, Matsumoto G, Kim S, Gu W, Devlin JP, Silverman RB, Morimoto RI; Celastrols as inducers of the heat shock response and cytoprotection. J Biol Chem (2004) 279(53):56053-56060. (Pubitemid 40066613)
60. Zhang YQ, Sarge KD: Celastrol inhibits polyglutamine aggregation and toxicity though induction of the heat shock response. J Mol Med (2007) 85(12);1421-1428.
61. 61. Kiaei M, Kipiani K, Petri S, Chen J, Calingasan NY, Beal MF: Celastrol blocks neuronal cell death and extends life in transgenic mouse model of amyotrophic lateral sclerosis. Neurodegener Dis (2005) 2(5): 246-254. (Pubitemid 43805908)
62. Kalmar B, Greensmith L: Activation of the heat shock response in a primary cellular model of motoneuron neurodegeneration-evidence for neuroprotective and neurotoxic effects. Cell Mol Biol Lett (2009) 14(2):319-335.
63. Trott A, West JD, Klaic L, Westerheide SD, Silverman RB, Morimoto RI, Morano KA; Activation of heat shock and antioxidant responses by the natural product celastrol: Transcriptional signatures of a thiol-targeted molecule. Mol Biol Cell (2008) 19(3);1104-1112.
64. Yan D, Saito K, Ohmi Y, Fujie N, Ohtsuka K: Paeoniflorin, a novel heat shock protein-inducing compound. Cell Stress Chaperones (2004) 9(4);378-389.
65. 65. Nagayama SI, Jono H, Suzaki H, Sakai K, Tsuruya E, Yamatsu I, Isohama Y, Miyata T, Kai H; Carbenoxolone, a new inducer of heat shock protein 70. Life Sci (2001) 69(24);2867-2873. (Pubitemid 33030275)
66. 66. Vigh L, Literati PN, Horvath I, Torok Z, Balogh G, Glatz A, Kovacs E, Boros I, Ferdinandy P, Farkas B, Jaszlits L et al: Bimoclomol: A nontoxic, hydroxylamine derivative with stress protein-inducing activity and cytoprotective effects. Nat Med (1997) 3(10):1150-1154. (Pubitemid 27444460)
67. Polakowski JS, Wegner CD, Cox BF: Bimoclomol elevates heat shock protein 70 and cytoprotects rat neonatal cardiomyocytes. Eur J Pharmacol (2002) 435(1);73-77.
68. 68. Hargitai J, Lewis H, Boros I, Racz T, Fiser A, Kurucz I, Benjamin I, Vigh L, Penzes Z, Csermely P, Latchman DS; Bimoclomol, a heat shock protein co-inducer, acts by the prolonged activation of heat shock factor-1. Biochem Biophys Res Commun (2003) 307(3);689-695. (Pubitemid 36944988)
69. 69. Torok Z, Tsvetkova NM, Balogh G, Horvath I, Nagy E, Penzes Z, Hargitai J, Bensaude O, Csermely P, Crowe JH, Maresca B et al: Heat shock protein coinducers with no effect on protein denaturation specifically modulate the membrane lipid phase. Proc Natl Acad Sci USA (2003) 100(6):3131-3136. (Pubitemid 36356551)
70. 70. Kalmar B, Burnstock G, Vrbová G, Urbanics R, Csermely P, Greensmith L: Upregulation of heat shock proteins rescues motoneurones from axotomy-induced cell death in neonatal rats. Exp Neurol (2002) 176(1):87-97. (Pubitemid 34762108)
71. Kalmar B, Novoselov S, Gray A, Cheetham ME, Margulis B, Greensmith L: Late stage treatment with arimoclomol delays disease progression and prevents protein aggregation in the SOD1G93A mouse model of ALS. J Neurochem (2008) 107(2):339-350.
72. 72. Kieran D, Kalmar B, Dick JRT, Riddoch-Contreras J, Burnstock G, Greensmith L: Treatment with arimoclomol, a coinducer of heat shock proteins, delays disease progression in ALS mice. Nat Med (2004) 10(4);402-405. (Pubitemid 38508519)
73. Strokov IA, Manukhina EB, Bakhtina LY, Malyshev IY, Zoloev GK, Kazikhanova SI, Ametov AS: The function of endogenous protective systems in patients with insulin-dependent diabetes mellitus and polyneuropathy: Effect of antioxidant therapy. Bull Exp Biol Med (2000) 130(10) :986-990.
74. 74. Calabrese V, Colombrita C, Sultana R, Scapagnini G, Calvani M, Butterfield DA, Stella AMG: Redox modulation of heat shock protein expression by acetylcarnitine in aging brain: Relationship to antioxidant status and mitochondrial function. Antioxid Redox Signaling (2006) 8(3- 4):404-416. (Pubitemid 43741312)
75. 75. Abdul HM, Calabrese V, Calvani M, Butterfield DA; Acetyl-L-carnitineinduced up-regulation of heat shock proteins protects cortical neurons against amyloid-p peptide 1-42-mediated oxidative stress and neurotoxicity: Implications for Alzheimer's disease. J Neurosci Res (2006) 84(2); 398-408. (Pubitemid 44126292)
76. Roth E: Nonnutritive effects of glutamine. J Nutr (2008) 138(10);2025S-2031S.
77. Bongers T, Griffiths RD, McArdle A: Exogenous glutamine: The clinical evidence. Crit Care Med (2007) 35(9 Suppl);S545-S552.
78. 78. Jing L, Wu Q, Wang F: Glutamine induces heat-shock protein and protects against Escherichia coli lipopolysaccharideinduced vascular hyporeactivity in rats. Crit Care (2007) 11(2):34. (Pubitemid 46488579)
79. Singleton KD, Wischmeyer PE; Glutamine induces heat shock protein expression via O-glycosylation and phosphorylation of HSF-1 and Sp1. JPEN J Parenter Enteral Nutr (2008) 32(4): 371-376.
80. 80. Ziegler TR, Ogden LG, Singleton KD, Luo M, Fernandez-Estivariz C, Griffith DP, Galloway JR, Wischmeyer PE; Parenteral glutamine increases serum heat shock protein 70 in critically ill patients. Intensive Care Med (2005) 31(8);1079-1086. (Pubitemid 41125339)
81. 81. Tomisato W, Tsutsumi S, Tsuchiya T, Mizushima T: Geranylgeranylacetone protects guinea pig gastric mucosal cells from gastric stressor-induced necrosis by induction of heat-shock proteins. Biol Pharm Bull (2001) 24(8):887-891. (Pubitemid 32750312)
82. 82. Nagai Y, Fujiki M, Inoue R, Uchida S, Abe T, Kobayashi H, Cetinalp NE: Neuroprotective effect of geranylgeranylacetone, a noninvasive heat shock protein inducer, on cerebral infarction in rats. Neurosa Lett (2005) 374(3): 183-188. (Pubitemid 40128292)
83. 83. Nishida T, Matsura T, Nakada J, Togawa A, Kai M, Sumioka I, Minami Y, Inagaki Y, Ishibe Y, Ko H, Ohta Y et al: Geranylgeranylacetone protects against acetaminophen-induced hepatotoxicity by inducing heat shock protein 70. Toxicology (2006) 219(1-3):187-196. (Pubitemid 43120719)
84. 84. Mochida S, Matsura T, Yamashita A, Horie S, Ohata S, Kusumoto C, Nishida T, Minami Y, Inagaki Y, Ishibe Y, Nakada J et al: Geranylgeranylacetone ameliorates inflammatory response to lipopolysaccharide (LPS) in murine macrophages: Inhibition of LPS binding to the cell surface. J Clin Biochem Nutr (2007) 41(2); 115-123. (Pubitemid 47529307)
85. Sanbe A, Daicho T, Mizutani R, Endo T, Miyauchi N, Yamauchi J, Tanonaka K, Glabe C, Tanoue A: Protective effect of geranylgeranylacetone via enhancement of HspBS induction in desmin-related cardiomyopathy. PLoS One (2009) 4(4):e5351
86. 86. Otaka M, Yamamoto S, Ogasawara K, Takaoka Y, Noguchi S, Miyazaki T1 Nakai A, Odashima M, Matsuhashi T, Watanabe S, Itoh H; The induction mechanism of the molecular chaperone Hsp70 in the gastric mucosa by Geranylgeranylacetone (HSP-inducer). Biochem Biophys Res Commun (2007) 353(2);399-404. (Pubitemid 46014576)
87. 87. Putics A, Végh EM, Csermely P, Soti C: Resveratrol induces the heat-shock response and protects human cells from severe heat stress. Antioxid Redox Signal (2008) 10(1);65-76. (Pubitemid 350115992)
88. Yang J, Bridges K, Chen KY, Liu AY: Riluzole increases the amount of latent HSFl for an amplified heat shock response and cytoprotection. PLoS One (2008) 3(8);e2864.
89. Revill P, Mealy N, Serradell N, Rosa E, Bolos J: Elesclomol. Drugs Future (2008) 33(4);310-315.
90. 90. Buommino E, Schiraldi C, Baroni A, Paoletti I, Lamberti M, De Rosa M, Tufano MA; Ectoine from halophilic microorganisms induces the expression of Hsp70 and Hsp70B' in human keratinocytes modulating the proinflammatory response. Cell Stress Chaperones (2005) 10(3): 197-203. (Pubitemid 41813366)
91. 91. O'Sullivan JC, Yao X, Alam H, McCabe JT; Diazoxide, as a postconditioning and delayed preconditioning trigger, increases Hsp25 and Hsp70 in the central nervous system following combined cerebral stroke and hemorrhagic shock. J Neurotrauma (2007) 24(3):532-546. (Pubitemid 46598286)
92. BioAssay Summary: PubChem BioAssay IDs 1203 and 1252, Compound ID 998879. The Scripps Research Institute Molecular Screening Center, La Jolla, CA, USA (2009). pubchem.ncbi.nlm. nih.gov/assay/assay.cgi?aid=1203&loc=ea- ras#aDescription and pubchem. ncbi.nlm.nih.gov/assay/assay. cgi?aid= 1252&loc=ea- ras#aDescription
93. 93. Yokota SI, Kitahara M, Nagata K: Benzylidene lactam compound, KNK437, a novel inhibitor of acquisition of thermotolerance and heat shock protein induction in human colon carcinoma cells. Cancer Res (2000) 60(11):2942-2948. (Pubitemid 30395819)
94. Wu YC, Yen WY, Lee TC, Yih LH: Heat shock protein inhibitors, 17-DMAG and KNK437, enhance arsenic trioxide-induced mitotic apoptosis. Toxicol Appl Pharmacol (2009) 236(2);231-238.
95. 95. Westerheide SD, Kawahara TL, Orton K, Morimoto RI: Triptolide, an inhibitor of the human heat shock response that enhances stress-induced cell death. J Biol Chem (2006) 281(14): 9616-9622. (Pubitemid 43864680)
96. 96. Powers MV, Workman P; Inhibitors of the heat shock response: Biology and pharmacology. FEBS Lett (2007) 581(19):3758-3769. (Pubitemid 47081010)
97. 97. Wei YQ, Zhao X, Kariya Y, Fukata H, Teshigawara K, Uchida A: Induction of apoptosis by quercetin: Involvement of heat shock protein. Cancer Res (1994) 54(18):4952-4957. (Pubitemid 24289427)
98. Nagai N, Nakai A, Nagata K: Quercetin suppresses heat shock response by down regulation of HSF1. Biochem Biophys Res Commun (1995) 208(3); 1099-1105.
99. Wang RE, Kao JLF, Hilliard CA, Pandita RK, Roti Roti JL, Hunt CR, Taylor JS: Inhibition of heat shock induction of heat shock protein 70 and enhancement of heat shock protein 27 phosphorylation by quercetin derivatives. J Med Chem (2009) 52(7): 1912-1921.
100. 100. Garrido C, Brunet M, Didelot C, Zermati Y, Schmitt E, Kroemer G: Heat shock proteins 27 and 70: Anti-apoptotic proteins with tumorigenic properties. Cell Cycle (2006) 5(22); 2592-2601. (Pubitemid 44785803)
101. Powers MV, Clarke PA, Workman P: Death by chaperone: Hsp90, Hsp70 or both? Cell Cycle (2009) 8(4):518-526.
102. Didelot C, Schmitt E, Brunet M, Maingret L, Parcellier A, Garrido C: Heat shock proteins: Endogenous modulators of apoptotic cell death. Handb Exp Pharmacol (2006) (172);171-198.
103. Gehrmann M: Drug evaluation: STA-4783 - Enhancing taxane efficacy by induction of Hsp70. Curr Opin Investig Drugs (2006) 7(6): 574-580.
104. 104. Daugaard M, Rohde M, Jäättelä M: The heat shock protein 70 family: Highly homologous proteins with overlapping and distinct functions. FEBS Lett (2007) 581(19);3702-3710. (Pubitemid 47081009)
105. Brodsky JL, Chiosis G: Hsp70 molecular chaperones: Emerging roles in human disease and identification of small molecule modulators. Curr Top Med Chem (2006) 6(11): 1215-1225.
106. 106. Morano KA; New tricks for an old dog: The evolving world of Hsp70. Ann NY Acad Sci (2007) 1113:1-14. (Pubitemid 350015016)
107. 107. Liu Q, Hendrickson WA: Insights into Hsp70 chaperone activity from a crystal structure of the yeast Hsp110 Ssel. Cell (2007) 131(1):106-120. (Pubitemid 47498533)
108. 108. Wang X, Venable J, LaPointe P, Hutt DM, Koulov AV, Coppinger J, Gurkan C, Kellner W, Matteson J, Plutner H, Riordan JR et al: Hsp90 cochaperone Ahal downregulation rescues misfolding of CFTR in cystic fibrosis. Cell (2006) 127(4):803-815. (Pubitemid 44716255)
109. 109. Arndt V, Rogón C, Hoehfeld J: To be, or not to be - Molecular chaperones in protein degradation. Cell Mol Life Sci (2007) 64 (19-20);2525-2541. (Pubitemid 350035611)
110. 110. Slavotinek AM, Biesecker LG; Unfolding the role of chaperones and chaperonins in human disease. Trends Genet (2001) 17(9):528-535. (Pubitemid 32763051)
111. 111. Gregersen N, Brass P, Andresen BS, Pedersen CB, Corydon TJ, Bolund L: The role of chaperone-assisted folding and quality control in inborn errors of metabolism: Protein folding disorders. J Inherited Metab Dis (2001) 24(2); 189-212. (Pubitemid 32479415)
112. Tanzi RE, Bertram L; Twenty years of the Alzheimer's disease amyloid hypothesis: A genetic perspective. Cell (2005) 120(4):545-555.
113. Churcher I: Tau therapeutic strategies for the treatment of Alzheimer's disease. Curr Top Med Chem (2006) 6(6);579-595.
114. Uversky VN: Neuropathology, biochemistry, and biophysics of a-synuclein aggregation. J Neurochem (2007) 103(1):17-37.
115. Trojanowski JQ, Lee VM; Parkinson's disease and related synucleinopathies are a new class of nervous system amyloidoses. Neurotoxicology (2002) 23(4-5); 457-460.
116. 116. Imarisio S, Carmichael J, Korolchuk V, Chen CW, Salki S, Rose C, Krishna G, Davies JE, Ttofi E, Underwood BR, Rubinsztein DC; Huntington's disease: From pathology and genetics to potential therapies. Biochem J (2008) 412(2): 191-209. (Pubitemid 351758226)
117. 117. Panet-Raymond V, Gottlieb B, Beitel LK, Schipper H, Timiansky M, Pinsky L, Trifiro MA: Characterization of intracellular aggregates using fluorescently-tagged polyglutamineexpanded androgen receptor. Neurotox Res (2001) 3(3);259-275. (Pubitemid 32962888)
118. Paulson H, Subramony SH; Spinocerebellar ataxia 3-Machado- Joseph disease (SCA3). In; Genetics of Movement Disorders. Pulst SM (Ed), Elsevier BV, Amsterdam, the Netherlands (2003);57-69.
119. Soong BW, Paulson HL: Spinocerebellar ataxias: An update. Curr Opin Neurol (2007) 20(4): 438-446.
120. 120. Katsuno M, Banno H, Suzuki K, Takeuchi Y, Kawashima M, Tanaka F, Adachi H, Sobue G: Molecular genetics and biomarkers of polyglutamine diseases. Curr Mol Med (2008) 8(3):221-234. (Pubitemid 351982540)
121. 121. Li L, Hoelscher C: Common pathological processes in Alzheimer disease and type 2 diabetes: A review. Brain Res Rev (2007) 56(2);384-402. (Pubitemid 350166529)
122. 122. Mondóla P, Seru R, Damiano S, Santillo MR: A new perspective on the role of CuZn superoxide dismutase (SODl). Cent Eur J Biol (2007) 2(3):337-350. (Pubitemid 47245951)
123. 123. Campana V, Sarnataro D, Zurzolo C: The highways and byways of prion protein trafficking. Trends Cell Biol (2005) 15(2): 102-111. (Pubitemid 40188220)
124. Montagna P, Gambettl P, Cortelli P, Lugaresi E; Familial and sporadic fatal insomnia. Lancet Neurol (2003) 2(3); 167-176.
125. Chung CM, Connors LH, Benson MD, Walsh MT; Biophysical analysis of normal transthyretin: Implications for fibril formation in senile systemic amyloidosis. Amyloid (2001) 8(2): 75-83.
126. Blagosklonny MV: Loss of function and p53 protein stabilization. Oncogene (1997) 15(16):1889-1893.
127. 127. Yu Z, Sawkar AR, Whalen LJ, Wong CH, Kelly JW: Isofagomine- and 2,5-anhydro-2,5-imino-D-glucitol-based glucocerebrosidase pharmacological chaperones for Gaucher disease intervention. J Med Chem (2007) 50(1);94-100. (Pubitemid 46105502)
128. Zarate YA, Hopkin RJ: Fabry's disease. Lancet (2008) 372(9647);1427-1435.
129. Myerowitz R: Tay-Sachs disease-causing mutations and neutral polymorphisms in the Hex A gene. Hum Mutat (1997) 9(3):195-208.
130. 130. Mark BL, Mahuran DJ, Cherney MM, Zhao D, Knapp S, James MN; Crystal structure of human β-hexosaminidase B: Understanding the molecular basis of Sandhoff and Tay-Sachs disease. J Mol Biol (2003) 327 (5);1093-1109. (Pubitemid 36363715)
131. 131. Jesionek-Kupnicka D, Majchrowska A, Krawczyk J, Wendorff J, Barcikowska M, Lukaszek S, Liberski PP: Krabbe disease: An ultrastructural study of globoid cells and reactive astrocytes at the brain and optic nerves. Folia Neuropathol (1997) 35(3):155-162. (Pubitemid 127533690)
132. Van der Ploeg AT, Reuser AJJ: Pompe's disease. Lancet (2008) 372(9646):1342-1353.
133. Yoshida K, Oshima A, Sakuraba H, Nakano T, Yanagisawa N, Inui K, Okada S, Uyama E, Namba R, Kondo K et al: GM1 gangliosidosis in adults: Clinical and molecular analysis of 16 Japanese patients. Ann Neurol (1992) 31(3):328-332.
134. 134. Dubern B, Bisbis S, Talbaoui H, Le Beyec J, Tounian P, Lacorte JM, Clement K; Homozygous null mutation of the melanocortin-4 receptor and severe early-onset obesity. J Pediatr (2007) 150(6);613-617. (Pubitemid 46778923)
135. Gelman MS, Kopito RR: Cystic fibrosis: Premature degradation of mutant proteins as a molecular disease mechanism. Methods Mol Biol (2003) 232:27-37.
136. 136. Janovick JA, Maya-Nunez G, Conn PM; Rescue of hypogonadotropic hypogonadism-causing and manufactured GnRH receptor mutants by a specific protein-folding template: Misrouted proteins as a novel disease etiology and therapeutic target. J Clin Endocrinol Metab (2002) 87(7);3255- 3262. (Pubitemid 34816374)
137. 137. Morello JP, Salahpour A, Laperrière A, Bernier V, Arthus MF, Lonergan M, Petäjä-Repo U, Angers S, Morin D, Bichet DG, Bouvier M; Pharmacological chaperones rescue cell-surface expression and function of misfolded V2 vasopressin receptor mutants. J Clin Invest (2000) 105(7): 887-895. (Pubitemid 30203061)
138. Maury CP, Kere J, Tolvanen R, de la Chapelle A: Finnish hereditary amyloidosis is caused by a single nucleotide substitution in the gelsolin gene. FEBS Lett (1990) 276(1-2):75-77.
139. 139. Siomi MC, Zhang Y, Siomi H, Dreyfuss G: Specific sequences in the fragile X syndrome protein FMRl and the FXR proteins mediate their binding to 60S ribosomal subunits and the interactions among them. Mol Cell Biol (1996) 16(7):3825-3832. (Pubitemid 26200257)
140. 140. Heegaard NHH, Roepstorff P, Melberg SG, Nissen MH: Cleaved (12-microglobulin partially attains a conformation that has amyloidogenicfeatures. J Biol Chem (2002) 277(13);11184-11189. (Pubitemid 34952880)
141. Elieder M; Niemann-Pick disease. Pathol Res Pract (1989) 185(3);293-328.
142. 142. Fykse EM, Sletten K, Husby G, Cornwell GG 3rd; The primary structure of the variable region of an immunoglobulin IV light-chain amyloid-fibril protein (AL GIL). Biochem J (1988) 256(3);973-980. (Pubitemid 19021730)
143. Ingram VM: Sickle-cell anemia hemoglobin: The molecular biology of the first 'molecular disease' - the crucial importance of serendipity. Genetics (2004) 167(1); 1-7.
144. 144. Mirchev R, Ferrone FA; The structural link between polymerization and sickle cell disease. J Mol Biol (1997) 265(5):475-479. (Pubitemid 27113034)
145. 145. Benson MD, Liepnieks J, Uemichi T, Wheeler G, Correa R; Hereditary renal amyloidosis associated with a mutant fibrinogen a-chain. Nat Genet (1993) 3(3);252-255. (Pubitemid 23096562)
146. Buxbaum JN: The systemic amyloidoses. Curr Opin Rheumatol (2004) 16(1);67-75.
147. 147. Teckman JH, Qu D, Perlmutter DH: Molecular pathogenesis of liver disease in α1-antitrypsin deficiency. Hepatology (1996) 24(6);1504-1516. (Pubitemid 26399057)
148. Ekeowa UI, Gooptu B, Belorgey D, Hägglöf P, Karlsson-Li S, Miranda E, Pérez J, MacLeod I, Kroger H, Marciniak SJ, Crowther DC et al: al-Antitrypsin deficiency, chronic obstructive pulmonary disease and the serpinopathies. Clin Sci (2009) 116(12);837-850.
149. Belorgey D, Hagglof P, Karlsson-Li S, Lomas DA; Protein misfolding and the serpinopathies. Prion (2007) 1(1);15-20.
150. 150. Ghiso J, Pons-Estel B, Frangione B; Hereditary cerebral amyloid angiopathy: The amyloid fibrils contain a protein which is a variant of cystatin C, an inhibitor of lysosomal cysteine proteases. Biochem Biophys Res Commun (1986) 136(2);548-554. (Pubitemid 16075328)
151. Benvenga S, Facchiano A: Atrial natriuretic peptide and amyloidosis. J Intern Med (1995) 237(5); 525-526.
152. 152. Knecht A, de Beer FC, Präs M: Serum amyloid A protein in familial Mediterranean fever. Ann Intern Med (1985) 102(1): 71-72. (Pubitemid 15187662)
153. 153. Gertz MA, Skinner M, Sipe JD, Cohen AS, Kyle RA; Serum amyloid A protein and C-reactive protein in systemic amyloidosis. Clin Exp Rheumatol (1985) 3(4): 317-320. (Pubitemid 16209624)
154. Pepys MB, Hawkins PN, Booth DR, Vigushin DM, Tennent GA, Soutar AK, Totty N, Nguyen O, Blake CC, Terry CJ et al: Human lysozyme gene mutations cause hereditary systemic amyloidosis, Nature (1993) 362(6420);553-557.
155. 155. Benson MD, Dwulet FE, Madura D, Wheeler G: Amyloidosis related to a XIV immunoglobulin light chain protein. Scand J Immunol (1989) 29(2); 175-179. (Pubitemid 19068586)
156. Wang L, Kluve-Beckerman B, Takasugi K, Okuda Y, Uemichi T, Yamada T, Nakamura M, Benson MD: Human serum amyloid Al allele frequencies and their relationship to the development of reactive amyloidosis in American Caucasian and Japanese populations. In: Amyloid Amyloidosis 1998: Proceedings of the VIIIth International Symposium on Amyloidosis. Kyle RA, Gertz MA (Eds), Taylor & Francis, London, UK (1999);375-377.
157. 157. Khurana R, Agarwal A, Bajpal V K, Verma N, Sharma AK, Gupta RP, Madhusudan KP; Unraveling the amyloid associated with human medullary thyroid carcinoma. Endocrinology (2004) 145(12);5465-5470. (Pubitemid 39564585)
158. 158. Sittler A, Lurz R, Lueder G, Priller J, Lehrach H, Hayer-Hartl MK, Hartl FU, Wanker EE; Geldanamycin activates a heat shock response and inhibits huntingtin aggregation in a cell culture model of Huntington's disease. Hum Mol Genet (2001) 10(12);1307-1315. (Pubitemid 32600484)
159. Marcu MG, Schulte TW, Neckers L: Novobiocin and related coumarins and depletion of heat shock protein 90-dependent signaling proteins. J Natl Cancer Inst (2000) 92(3); 242-248.
160. 2 and its inhibition by intracellular glutathione. Biochem Pharmacol (1993) 45(12);2457-2464.
161. 161. Batulan Z, Nalbantoglu J, Durham HD; Nonsteroidal antiinflammatory drugs differentially affect the heat shock response in cultured spinal cord cells. Cell Stress Chaperones (2005) 10(3): 185-196. (Pubitemid 41813365)
162. Zhang T, Hamza A, Cao X, Wang B, Yu S, Zhan CG, Sun D; A novel Hsp90 inhibitor to disrupt Hsp90/Cdc37 complex against pancreatic cancer cells. Mol Cancer Ther (2008) 7(1):162-170.
163. Abdul HM, Butterfield DA: Involvement of PI3K/PKG/ERK1/2 signaling pathways in cortical neurons to trigger protection by cotreatment of acetyl-L-carnitine and a-lipoic acid against HNE-mediated oxidative stress and neurotoxicity: Implications for Alzheimer's disease. Free Radie Biol Med (2007) 42(3);371-384.
164. Ehrenfried JA, Chen J, Li J, Evers BM: Glutamine-mediated regulation of heat shock protein expression in intestinal cells. Surgery (1995) 118(2);352-356.
165. 165. Hirakawa T, Rokutan K, Nikawa T, Kishi K; Geranylgeranylacetone induces heat shock proteins in cultured guinea pig gastric mucosal cells and rat gastric mucosa. Gastroenterology (1996) 111(2);345-357. (Pubitemid 26165713)
166. Nakanoma T, Ueno M, lida M, Hirata R, Deguchi N: Effects of quercetin on the heat-induced cytotoxicity of prostate cancer cells. Int J Urol (2001) 8(11):623-630.