Trehalose alleviates polyglutamine-mediated pathology in a mouse model of Huntington disease (original) (raw)

• Zoghbi, H.Y. & Orr, H.T. Glutamine repeats and neurodegeneration. Annu. Rev. Neurosci. 23, 217–247 (2000).
  Article CAS Google Scholar
• Scherzinger, E. et al. Huntingtin-encoded polyglutamine expansions form amyloid-like protein aggregates in vitro and in vivo. Cell 90, 549–558 (1997).
  Article CAS Google Scholar
• Davies, S.W. et al. Formation of neuronal intranuclear inclusions underlies the neurological dysfunction in mice transgenic for the HD mutation. Cell 90, 537–548 (1997).
  Article CAS Google Scholar
• Warrick, J.M. et al. Expanded polyglutamine protein forms nuclear inclusions and causes neural degeneration in Drosophila. Cell 93, 939–949 (1998).
  Article CAS Google Scholar
• Lunkes, A. & Mandel, J.L. A cellular model that recapitulates major pathogenic steps of Huntington's disease. Hum. Mol. Genet. 7, 1355–1361 (1998).
  Article CAS Google Scholar
• DiFiglia, M. et al. Aggregation of huntingtin in neuronal intranuclear inclusions and dystrophic neurites in brain. Science 277, 1990–1993 (1997).
  Article CAS Google Scholar
• Michalik, A. & Van Broeckhoven, C. Pathogenesis of polyglutamine disorders: aggregation revisited. Hum. Mol. Genet. 12, R173–R186 (2003).
  Article CAS Google Scholar
• Mccampbell, A. & Fischbeck, K.H. Polyglutamine and CBP: fatal attraction? Nat. Med. 7, 528–530 (2001).
  Article CAS Google Scholar
• Ross, C.A., Poirier, M.A., Wanker, E.E. & Amzel, M. Polyglutamine fibrillogenesis: the pathway unfolds. Proc. Natl. Acad. Sci. USA 100, 1–3 (2003).
  Article CAS Google Scholar
• Bates, G. Huntingtin aggregation and toxicity in Huntington's disease. Lancet 361, 1642–1644 (2003).
  Article CAS Google Scholar
• Ona, V.O. et al. Inhibition of caspase-1 slows disease progression in a mouse model of Huntington's disease. Nature 399, 263–267 (1999).
  Article CAS Google Scholar
• Cummings, C.J. et al. Chaperone suppression of aggregation and altered subcellular proteasome localization imply protein misfolding in SCA1. Nat. Genet. 19, 148–154 (1998).
  Article CAS Google Scholar
• Jana, N.R., Tanaka, M., Wang, G.H. & Nukina, N. Polyglutamine length–dependent interaction of Hsp40 and Hsp70 family chaperones with truncated N-terminal huntingtin: their role in suppression of aggregation and cellular toxicity. Hum. Mol. Genet. 9, 2009–2018 (2000).
  Article CAS Google Scholar
• Shimohata, T. et al. Expanded polyglutamine stretches interact with TAFII130, interfering with CREB-dependent transcription. Nat. Genet. 26, 29–36 (2000).
  Article CAS Google Scholar
• Perez, M.K. et al. Recruitment and the role of nuclear localization in polyglutamine-mediated aggregation. J. Cell. Biol. 143, 1457–1470 (1998).
  Article CAS Google Scholar
• Chen, M. et al. Minocycline inhibits caspase-1 and caspase-3 expression and delays mortality in a transgenic mouse model of Huntington disease. Nat. Med. 6, 797–801 (2000).
  Article CAS Google Scholar
• Ferrante, R.J. et al. Neuroprotective effects of creatine in a transgenic mouse model of Huntington's disease. J. Neurosci. 20, 4389–4397 (2000).
  Article CAS Google Scholar
• Hockly, E. et al. Suberoylanilide hydroxamic acid, a histone deacetylase inhibitor, ameliorates motor deficits in a mouse model of Huntington's disease. Proc. Natl. Acad. Sci. USA 100, 2041–2046 (2003).
  Article CAS Google Scholar
• Sanchez, I., Mahlke, C. & Yuan, J. Pivotal role of oligomerization in expanded polyglutamine neurodegenerative disorders. Nature 421, 373–379 (2003).
  Article CAS Google Scholar
• Hughes, R.E. & Olson, J.M. Therapeutic opportunities in polyglutamine disease. Nat. Med. 7, 419–423 (2001).
  Article CAS Google Scholar
• Heiser, V. et al. Inhibition of huntingtin fibrillogenesis by specific antibodies and small molecules: implication for Huntington's disease therapy. Proc. Natl. Acad. Sci. USA 97, 6739–6744 (2000).
  Article CAS Google Scholar
• Heiser, V. et al. Identification of benzothiazoles as potential polyglutamine aggregation inhibitors of Huntington's disease by using an automated filter retardation assay. Proc. Natl. Acad. Sci. USA 99, 16400–16406 (2002).
  Article CAS Google Scholar
• Tanaka, M., Morishima, I., Akagi, T., Hashikawa, T. & Nukina, N. Intra- and intermolecular β-pleated sheet formation in glutamine-repeat inserted myoglobin as a model for polyglutamine diseases. J. Biol. Chem. 276, 45470–45475 (2001).
  Article CAS Google Scholar
• Tanaka, M. et al. The effects of aggregation-inducing motifs on amyloid formation of model proteins related to neurodegenerative diseases. Biochemistry 41, 10277–10286 (2002).
  Article CAS Google Scholar
• Nagai, Y. et al. Inhibition of polyglutamine protein aggregation and cell death by novel peptides identified by phage display screening. J. Biol. Chem. 275, 10437–10442 (2000).
  Article CAS Google Scholar
• Luo, Y. & Baldwin, R.L. Trifluoroethanol stabilizes the pH 4 folding intermediate of sperm whale apomyoglobin. J. Mol. Biol. 279, 49–57 (1998).
  Article CAS Google Scholar
• Wang, G.H. et al. Caspase activation during apoptotic cell death induced by expanded polyglutamine in N2a cells. Neuroreport 10, 2435–2438 (1999).
  Article CAS Google Scholar
• Guo, N., Puhlev, I., Brown, D.R., Mansbridge, J. & Levine, F. Trehalose expression confers desiccation tolerance on human cells. Nat. Biotechnol. 18, 168–171 (2000).
  Article CAS Google Scholar
• Mangiarini, L. et al. Exon 1 of the HD gene with an expanded CAG repeat is sufficient to cause a progressive neurological phenotype in transgenic mice. Cell 87, 493–506 (1996).
  Article CAS Google Scholar
• Sathasivam, K. et al. Formation of polyglutamine inclusions in non-CNS tissue. Hum. Mol. Genet. 8, 813–822 (1999).
  Article CAS Google Scholar
• Poirier, M.A. et al. Huntingtin spheroids and protofibrils as precursors in polyglutamine fibrilization. J. Biol. Chem. 277, 41032–41037 (2002).
  Article CAS Google Scholar
• Azzouz, M. et al. Increased motoneuron survival and improved neuromuscular function in transgenic ALS mice after intraspinal injection of an adeno-associated virus encoding Bcl-2. Hum. Mol. Genet. 9, 803–811 (2000).
  Article CAS Google Scholar
• Hurlbert, M.S. et al. Mice transgenic for an expanded CAG repeat in the Huntington's disease gene develop diabetes. Diabetes 48, 649–651 (1999).
  Article CAS Google Scholar
• Farrer, A. Diabetes mellitus in Huntington's disease. Clin. Genet. 27, 62–67 (1985).
  Article CAS Google Scholar
• Singer, M.A. & Lindquist, S. Multiple effects of trehalose on protein folding in vitro and in vivo. Mol. Cell 1, 639–648 (1998).
  Article CAS Google Scholar
• Kazantsev, A. et al. A bivalent Huntingtin binding peptide suppresses polyglutamine aggregation and pathogenesis in Drosophila. Nat. Genet. 30, 367–376 (2002).
  Article CAS Google Scholar
• Yang, W., Dunlap, J.R., Andrews, R.B. & Wetzel, R. Aggregated polyglutamine peptides delivered to nuclei are toxic to mammalian cells. Hum. Mol. Genet. 11, 2905–2917 (2002).
  Article CAS Google Scholar
• Wellington, C.L. et al. Caspase cleavage of gene products associated with triplet expansion disorders generates truncated fragments containing the polyglutamine tract. J. Biol. Chem. 273, 9158–9167 (1998).
  Article CAS Google Scholar
• Kandror, O., DeLeon, A. & Goldberg, A.L. Trehalose synthesis is induced upon exposure of Escherichia coli to cold and is essential for viability at low temperature. Proc. Natl. Acad. Sci. USA 99, 9727–9732 (2002).
  Article CAS Google Scholar