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Original Article

Identification of compounds protective against G93A-SOD1 toxicity for the treatment of amyotrophic lateral sclerosis

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Pages 87-96 | Received 15 Jun 2010, Accepted 07 Sep 2010, Published online: 12 Nov 2010

References

  • Brown RWJ. The Motor Neuron Diseases. Fauchi AS, . Harrison's Principles of Internal Medicine. New York: McGraw-Hill; 1998. 2368–71.
  • Bensimon G, Lacomblez L, Meininger V. A controlled trial of riluzole in amyotrophic lateral sclerosis. ALS/Riluzole Study Group. N Engl J Med. 1994;330:585–91.
  • Francis K, Bach JR, DeLisa JA. Evaluation and rehabilitation of patients with adult motor neuron disease. Arch Phys Med Rehabil. 1999;80:951–63.
  • Miller RG, Mitchell JD, Lyon M, Moore DH. Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND). Amyotroph Lateral Scler Other Motor Neuron Disord. 2003;4:191–206.
  • Bruijn LI, Miller TM, Cleveland DW. Unraveling the mechanisms involved in motor neuron degeneration in ALS. Annu Rev Neurosci. 2004;27:723–49.
  • Kwiatkowski TJ Jr, Bosco DA, Leclerc AL, Tamrazian E, van der Burg CR, Russ C, . Mutations in the FUS/TLS gene on chromosome 16 cause familial amyotrophic lateral sclerosis. Science. 2009;323:1205–8.
  • Vance C, Rogelj B, Hortobagyi T, de Vos KJ, Nishimura AL, Sreedharan J, . Mutations in FUS, an RNA processing protein, cause familial amyotrophic lateral sclerosis type 6. Science. 2009;323:1208–11.
  • Ryu H, Smith K, Camelo SI, Carreras I, Lee J, Iglesias AH, . Sodium phenylbutyrate prolongs survival and regulates expression of anti-apoptotic genes in transgenic amyotrophic lateral sclerosis mice. J Neurochem. 2005;93:1087–98.
  • Rothstein JD. Current hypotheses for the underlying biology of amyotrophic lateral sclerosis. Ann Neurol. 2009;65:S3–9.
  • Brown RH Jr, Robberecht W. Amyotrophic lateral sclerosis: pathogenesis. Semin Neurol. 2001;21:131–9.
  • Gaudette M, Hirano M, Siddique T. Current status of SOD1 mutations in familial amyotrophic lateral sclerosis. Amyotroph Lateral Scler Other Motor Neuron Disord. 2000;1:83–9.
  • Arai T, Hasegawa M, Akiyama H, Ikeda K, Nonaka T, Mori H, . TDP-43 is a component of ubiquitin-positive tau-negative inclusions in frontotemporal lobar degeneration and amyotrophic lateral sclerosis. Biochem Biophys Res Commun. 2006;351:602–11.
  • Mackenzie IR, Bigio EH, Ince PG, Geser F, Neumann M, Cairns NJ, . Pathological TDP-43 distinguishes sporadic amyotrophic lateral sclerosis from amyotrophic lateral sclerosis with SOD1 mutations. Ann Neurol. 2007;61: 427–34.
  • Sreedharan J, Blair IP, Tripathi VB, Hu X, Vance C, Rogelj B, . TDP-43 mutations in familial and sporadic amyotrophic lateral sclerosis. Science. 2008;319: 1668–72.
  • Strong MJ, Volkening K, Hammond R, Yang W, Strong W, Leystra-Lantz C, . TD-P43 is a human low molecular weight neurofilament (hNFL) mRNA-binding protein. Mol Cell Neurosci. 2007;35:320–7.
  • Yokoseki A, Shiga A, Tan CF, Tagawa A, Kaneko H, Koyama A, . TDP-43 mutation in familial amyotrophic lateral sclerosis. Ann Neurol. 2008;63:538–42.
  • Traynor BJ, Bruijn L, Conwit R, Beal F, O'Neill G, Fagan SC, . Neuroprotective agents for clinical trials in ALS: a systematic assessment. Neurology. 2006;67: 20–7.
  • Wong PC, Cai H, Borchelt DR, Price DL. Genetically engineered mouse models of neurodegenerative diseases. Nat Neurosci. 2002;5:633–9.
  • Cudkowicz ME, Andres PL, Macdonald SA, Bedlack RS, Choudry R, Brown RH Jr, . Phase II study of sodium phenylbutyrate in ALS. Amyotroph Lateral Scler. 2009;10: 99–106.
  • Ryu H, Ferrante RJ. Translational therapeutic strategies in amyotrophic lateral sclerosis. Mini Rev Med Chem. 2007;7:141–50.
  • Matsumoto G, Kim S, Morimoto RI. Huntingtin and mutant SOD1 form aggregate structures with distinct molecular properties in human cells. J Biol Chem. 2006;281: 4477–85.
  • Matsumoto G, Stojanovic A, Holmberg CI, Kim S, Morimoto RI. Structural properties and neuronal toxicity of amyotrophic lateral sclerosis-associated Cu/Zn superoxide dismutase 1 aggregates. J Cell Biol. 2005;171:75–85.
  • Lee DH, Goldberg AL. Proteasome inhibitors: valuable new tools for cell biologists. Trends Cell Biol. 1998;8:397–403.
  • Morimoto RI. Proteotoxic stress and inducible chaperone networks in neurodegenerative disease and aging. Genes Dev. 2008;22:1427–38.
  • Zhang JH, Chung TD, Oldenburg KR. A simple statistical parameter for use in evaluation and validation of high-throughput screening assays. J Biomol Screen. 1999;4:67–73.
  • Prudencio M, Hart PJ, Borchelt DR, Andersen PM. Variation in aggregation propensities among ALS-associated variants of SOD1: correlation to human disease. Hum Mol Genet. 2009;18:3217–26.
  • Corcoran LJ, Mitchison TJ, Liu Q. A novel action of histone deacetylase inhibitors in a protein aggresome disease model. Curr Biol. 2004;14:488–92.
  • Kabashi E, Valdmanis PN, Dion P, Spiegelman D, McConkey BJ, van de Velde C, . TARDBP mutations in individuals with sporadic and familial amyotrophic lateral sclerosis. Nat Genet. 2008;40:572–4.
  • Benatar M. Lost in translation: treatment trials in the SOD1 mouse and in human ALS. Neurobiol Dis. 2007;26:1–13.
  • Scott S, Kranz JE, Cole J, Lincecum JM, Thompson K, Kelly N, . Design, power, and interpretation of studies in the standard murine model of ALS. Amyotroph Lateral Scler. 2008;9:4–15.
  • Karch CM, Prudencio M, Winkler DD, Hart PJ, Borchelt DR. Role of mutant SOD1 disulfide oxidation and aggregation in the pathogenesis of familial ALS. Proc Natl Acad Sci U S A. 2009;106:7774–9.
  • Wang J, Farr GW, Zeiss CJ, Rodriguez-Gil DJ, Wilson JH, Furtak K, . Progressive aggregation despite chaperone associations of a mutant SOD1-YFP in transgenic mice that develop ALS. Proc Natl Acad Sci U S A. 2009;106:1392–7.
  • Wegorzewska I, Bell S, Cairns NJ, Miller TM, Baloh RH. TDP-43 mutant transgenic mice develop features of ALS and frontotemporal lobar degeneration. Proc Natl Acad Sci U S A. 2009;106:18809–14.
  • Wils H, Kleinberger G, Janssens J, Pereson S, Joris G, Cuijt I, . TDP-43 transgenic mice develop spastic paralysis and neuronal inclusions characteristic of ALS and frontotemporal lobar degeneration. Proc Natl Acad Sci U S A. 2010;107:3858–63.
  • Heemskerk J, Tobin AJ, Bain LJ. Teaching old drugs new tricks. Meeting of the Neurodegeneration Drug Screening Consortium, 7–8 April 2002, Washington, DC, USA. Trends Neurosci. 2002;25:494–6.
  • Lipinski CA, Lombardo F, Dominy BW, Feeney PJ. Experimental and computational approaches to estimate solubility and permeability in drug discovery and development settings. Advanced Drug Delivery Reviews. 1997;23:3–25.

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