ASC-J9 ameliorates spinal and bulbar muscular atrophy phenotype via degradation of androgen receptor

Nature Medicine

Volumn 13, Issue 3, 2007, Pages 348-353

  Yang, Zhiming ^a,b   Chang, Yu Jia ^a,c   Yu, I Chen ^a   Yeh, Shuyuan ^a   Wu, Cheng Chia ^a,c   Miyamoto, Hiroshi ^a   Merry, Diane E ^d   Sobue, Gen ^e   Chen, Lu Min ^a,f   Chang, Shu Shi ^a,f   Chang, Chawnshang ^a  

^a UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

^b ZHEJIANG UNIVERSITY   (China)

^c TAIPEI MEDICAL UNIVERSITY   (Taiwan)

^d THOMAS JEFFERSON UNIVERSITY   (United States)

^e NAGOYA UNIVERSITY   (Japan)

^f CHINA MEDICAL UNIVERSITY   (Taiwan)

Author keywords

[No Author keywords available]

Indexed keywords

5 HYDROXY 1,7 BIS(3,4 DIMETHOXYPHENYL) 1,4,6 HEPTATRIEN 3 ONE; ANDROGEN RECEPTOR; ANTIANDROGEN; POLYGLUTAMINE; TESTOSTERONE; UNCLASSIFIED DRUG; VASCULOTROPIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL CULTURE; CELL SURVIVAL; CONTROLLED STUDY; FEMALE; FERTILITY; KENNEDY DISEASE; MALE; MOUSE; MUSCLE ATROPHY; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; SEXUAL FUNCTION; TESTOSTERONE BLOOD LEVEL;

ANIMALS; CELL LINE; CERCOPITHECUS AETHIOPS; COS CELLS; CURCUMIN; DISEASE MODELS, ANIMAL; FEMALE; MALE; MICE; MICE, TRANSGENIC; MUSCULAR ATROPHY, SPINAL; PHENOTYPE; RECEPTORS, ANDROGEN;

MUS;

EID: 33847738994     PISSN: 10788956     EISSN: 1546170X     Source Type: Journal    
DOI: 10.1038/nm1547     Document Type: Article

References (37)

1. Kennedy, W.R., Alter, M. & Sung, J.H. Progressive proximal spinal and bulbar muscular atrophy of late onset: a sex-linked recessive trait. Neurology 50, 583-593 (1998).
2. La Spada, A.R., Wilson, E.M., Lubahn, D.B., Harding, A.E. & Fischbeck, K.H. Androgen receptor gene mutations in X-linked spinal and bulbar muscular atrophy. Nature 352, 77-79 (1991).
3. Ringel, S.P., Lava, N.S., Treihaft, M.M., Lubs, M.L. & Lubs, H.A. Late-onset X-linked recessive spinal and bulbar muscular atrophy. Muscle Nerve 1, 297-307 (1978).
4. Sobue, G. et al. X-linked recessive bulbospinal neuronopathy. A clinicopathological study. Brain 112, 209-232 (1989).
5. Katsuno, M. et al. Testosterone reduction prevents phenotypic expression in a transgenic mouse model of spinal and bulbar muscular atrophy. Neuron 35, 843-854 (2002).
6. Li, M. et al. Nuclear inclusions of the androgen receptor protein in spinal and bulbar muscular atrophy. Ann. Neurol. 44, 249-254 (1998).
7. Chang, C.S., Kokontis, J. & Liao, S.T. Molecular cloning of human and rat complementary DNA encoding androgen receptors. Science 240, 324-326 (1988).
8. Heinlein, C.A. & Chang, C. Androgen receptor in prostate cancer. Endocr. Rev. 25, 276-308 (2004).
9. Yeh, S. & Chang, C. Cloning and characterization of a specific coactivator, ARA70, for the androgen receptor in human prostate cells. Proc. Natl. Acad. Sci. USA 93, 5517-5521 (1996).
10. Heinlein, C.A. & Chang, C. Androgen receptor (AR) coregulators: an overview. Endocr. Rev. 23, 175-200 (2002).
11. McCampbell, A. & Fischbeck, K.H. Polyglutamine and CBP: fatal attraction? Nat. Med. 7, 528-530 (2001).
12. Sherman, M.Y. & Goldberg, A.L. Cellular defenses against unfolded proteins: a cell biologist thinks about neurodegenerative diseases. Neuron 29, 15-32 (2001).
13. Ross, C.A. Polyglutamine pathogenesis: emergence of unifying mechanisms for Huntington's disease and related disorders. Neuron 35, 819-822 (2002).
14. Merry, D.E., Kobayashi, Y., Bailey, C.K., Taye, A.A. & Fischbeck, K.H. Cleavage, aggregation and toxicity of the expanded androgen receptor in spinal and bulbar muscular atrophy. Hum. Mol. Genet. 7, 693-701 (1998).
15. Tarlac, V. & Storey, E. Role of proteolysis in polyglutamine disorders. J. Neurosci. Res. 74, 406-416 (2003).
16. Mandrusiak, L.M. et al. Transglutaminase potentiates ligand-dependent proteasome dysfunction induced by polyglutamine-expanded androgen receptor. Hum. Mol. Genet. 12, 1497-1506 (2003).
17. Beauchemin, A.M. et al. Cytochrome c oxidase subunit Vb interacts with human androgen receptor: a potential mechanism for neurotoxicity in spinobulbar muscular atrophy. Brain Res. Bull. 56, 285-297 (2001).
18. Katsuno, M. & Sobue, G. Polyglutamine diminishes VEGF; passage to motor neuron death? Neuron 41, 677-679 (2004).
19. Katsuno, M. et al. Leuprorelin rescues polyglutamine-dependent phenotypes in a transgenic mouse model of spinal and bulbar muscular atrophy. Nat. Med. 9, 768-773 (2003).
20. Minamiyama, M. et al. Sodium butyrate ameliorates phenotypic expression in a transgenic mouse model of spinal and bulbar muscular atrophy. Hum. Mol. Genet. 13, 1183-1192 (2004).
21. Chevalier-Larsen, E.S. et al. Castration restores function and neurofilament alterations of aged symptomatic males in a transgenic mouse model of spinal and bulbar muscular atrophy. J. Neurosci. 24, 4778-4786 (2004).
22. Ohtsu, H. et al. Antitumor agents. 217. Curcumin analogues as novel androgen receptor antagonists with potential as anti-prostate cancer agents. J. Med. Chem. 45, 5037-5042 (2002).
23. Walcott, J.L. & Merry, D.E. Ligand promotes intranuclear inclusions in a novel cell model of spinal and bulbar muscular atrophy. J. Biol. Chem. 277, 50855-50859 (2002).
24. Sopher, B.L. et al. Androgen receptor YAC transgenic mice recapitulate SBMA motor neuronopathy and implicate VEGF164 in the motor neuron degeneration. Neuron 41, 687-699 (2004).
25. Waza, M. et al. 17-AAG, an Hsp90 inhibitor, ameliorates polyglutamine-mediated motor neuron degeneration. Nat. Med. 11, 1088-1095 (2005).
26. Goetz, M.P., Toft, D.O., Ames, M.M. & Erlichman, C. The Hsp90 chaperone complex as a novel target for cancer therapy. Ann. Oncol. 14, 1169-1176 (2003).
27. Solit, D.B. et al. 17-Allylamino-17-demethoxygeldanamycin induces the degradation of androgen receptor and HER-2/neu and inhibits the growth of prostate cancer xenografts. Clin. Cancer Res. 8, 986-993 (2002).
28. Barent, R.L. et al. Analysis of FKBP51/FKBP52 chimeras and mutants for Hsp90 binding and association with progesterone receptor complexes. Mol. Endocrinol. 12, 342-354 (1998).
29. Smith, D.F. et al. Progesterone receptor structure and function altered by geldanamycin, an hsp90-binding agent. Mol. Cell. Biol. 15, 6804-6812 (1995).
30. Hostein, I., Robertson, D., DiStefano, F., Workman, P. & Clarke, P.A. Inhibition of signal transduction by the Hsp90 inhibitor 17-allylamino-17- demethoxygeldanamycin results in cytostasis and apoptosis. Cancer Res. 61, 4003-4009 (2001).
31. Banerji, U. et al. Phase I pharmacokinetic and pharmacodynamic study of 17-allylamino, 17-demethoxygeldanamycin in patients with advanced malignancies. J. Clin. Oncol. 23, 4152-4161 (2005).
32. Price, J.T. et al. The heat shock protein 90 inhibitor, 17-allylamino-17-demethoxygeldanamycin, enhances osteoclast formation and potentiates bone metastasis of a human breast cancer cell line. Cancer Res. 65, 4929-4938 (2005).
33. Thin, T.H. et al. Mutations in the helix 3 region of the androgen receptor abrogate ARA70 promotion of 17beta-estradiol-induced androgen receptor transactivation. J. Biol. Chem. 277, 36499-36508 (2002).
34. Wang, L. et al. Suppression of androgen receptor-mediated transactivation and cell growth by the glycogen synthase kinase 3 beta in prostate cells. J. Biol. Chem. 279, 32444-32452 (2004).
35. Garden, G.A. et al. Polyglutamine-expanded ataxin-7 promotes non-cell-autonomous purkinje cell degeneration and displays proteolytic cleavage in ataxic transgenic mice. J. Neurosci. 22, 4897-4905 (2002).
36. Adachi, H. et al. Transgenic mice with an expanded CAG repeat controlled by the human AR promoter show polyglutamine nuclear inclusions and neuronal dysfunction without neuronal cell death. Hum. Mol. Genet. 10, 1039-1048 (2001).
37. Terao, S. et al. Age-related changes in human spinal ventral horn cells with special reference to the loss of small neurons in the intermediate zone: a quantitative analysis. Acta Neuropathol. (Berl.) 92, 109-114 (1996).