Development of Molecular Targeted Disease Modifying Therapy for Polyglutamine Diseases

• Project/Area Number: 21229011
• Research Category: Grant-in-Aid for Scientific Research (S)
• Allocation Type: Single-year Grants
• Research Field: Neurology
• Research Institution: Nagoya University
• Principal Investigator: SOBUE Gen 名古屋大学, 医学(系)研究科(研究院), 教授 (20148315)
• Co-Investigator(Kenkyū-buntansha): TANAKA Fumiaki 横浜市立大学, 医学研究科, 教授 (30378012) ; ADACHI Hiroaki 名古屋大学, 医学系研究科, 寄附講座准教授 (40432257) ; KATSUNO Masahisa 名古屋大学, 医学系研究科, 准教授 (50402566) ; IIJIMA Masahiro 名古屋大学, 医学系研究科, 寄附講座講師 (40437041) ; KOIKE Haruki 名古屋大学, 医学部附属病院, 病院講師 (80378174)
• Co-Investigator(Renkei-kenkyūsha): OKADA Youhei 慶應義塾大学, 医学部, 講師 (30383714) ; OKANO Hideyuki 慶應義塾大学, 医学部, 教授 (60160694) ; TANAKA Keiji 東京都医学総合研究所, 所長 (10108871)
• Project Period (FY): 2009-05-11 – 2014-03-31
• Project Status: Completed (Fiscal Year 2013)
• Budget Amount: ¥158,730,000 (Direct Cost: ¥122,100,000、Indirect Cost: ¥36,630,000); Fiscal Year 2013: ¥29,510,000 (Direct Cost: ¥22,700,000、Indirect Cost: ¥6,810,000); Fiscal Year 2012: ¥29,120,000 (Direct Cost: ¥22,400,000、Indirect Cost: ¥6,720,000); Fiscal Year 2011: ¥29,120,000 (Direct Cost: ¥22,400,000、Indirect Cost: ¥6,720,000); Fiscal Year 2010: ¥29,120,000 (Direct Cost: ¥22,400,000、Indirect Cost: ¥6,720,000); Fiscal Year 2009: ¥41,860,000 (Direct Cost: ¥32,200,000、Indirect Cost: ¥9,660,000)
• Keywords: ポリグルタミン病 / 球脊髄性筋萎縮症 / 神経変性 / 細胞周期 / JNK / 運動ニューロン / ユビキチン-プロテアゾーム系 / オートファジー / 分子シャペロン / )球脊髄性筋萎縮症 / ポリグルタミン / ユビキチン / プロテアソーム / ユビチキン / 転写障害 / セロトニン受容体アゴニスト / calcitonin/calcitonin-related polypeptide(CGRP1) / TGF-β / calcitonin / calcitonin-related polypeptide(CGRP1)

Research Abstract

Spinal and bulbar muscular atrophy (SBMA)is a motor neuron disease caused by an expansion of CAG trinucleotide expansion with in the androgen receptor (AR) gene. Nuclear accumulation of mutant AR triggers the degeneration of motor neurons in SBMA. Our results indicate that autophagy plays a protective role in SBMA, and that a small compound activating both molecular chaperones and autophagy suppresses the motor neuron degeneration in this disease. We also demonstrated that exogenous supplementation of miRNA that is up-regulated in SBMA mice attenuates mutant AR-mediated motor neuron degeneration.

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Research Products

• [Journal Article] Anti-androgen flutamide protects male mice from androgen-dependent toxicity in three models of spinal bulbar muscular atrophy (2014)
  • Author(s): Renier KJ, Troxell-Smith SM, Johansen JA, Katsuno M, Adachi H, Sobue G, Chua JP, Sun Kim H, Lieberman AP, Breedlove SM, Jordan CL
  • Journal Title: Endocrinology Volume: (in press)
  • Peer Reviewed
• [Journal Article] Paeoniflorin eliminates a mutant AR via NF-YA-dependent proteolysis in spinal and bulbar muscular atrophy (2014)
  • Author(s): Tohnai G, Adachi H, Katsuno M, Doi H, Matsumoto S, Kondo N, Miyazaki Y, Iida M, Nakatsuji H, Qiang Q, Ding Y, Watanabe H, Yamamoto M, Ohtsuka K, Sobue G
  • Journal Title: Hum Mol Genet Volume: (in press)
  • Peer Reviewed
• [Journal Article] Transcriptional activation of TFEB/ZKSCAN3 target genes underlies enhanced autophagy in spinobulbar muscular atrophy (2014)
  • Author(s): Chua JP, Reddy SL, Merry DE, Adachi H, Katsuno M, Sobue G, Robins DM, Lieberman AP
  • Journal Title: Hum Mol Genet Volume: (Epub ahead of print) Issue: 5 Pages: 1376-1386
  • DOI: 10.1093/hmg/ddt527
  • Peer Reviewed
• [Journal Article] Tongue pressure as a novel biomarker of spinal and bulbar muscular atrophy (SBMA) (2014)
  • Author(s): Mano T, Katsuno M, Banno H, Suzuki K, Suga N, Hashizume A, Araki A, Watanabe H, Tanaka S, Yamamoto M, Sobue G
  • Journal Title: Neurology Volume: 82 Issue: 3 Pages: 255-262
  • DOI: 10.1212/wnl.0000000000000041
  • Peer Reviewed
• [Journal Article] p62/SQSTM1 differentially removes the toxic mutant androgen receptor via autophagy and inclusion formation in a spinal and bulbar muscular atrophy mouse model (2013)
  • Author(s): Doi H, Adachi H, Katsuno M, Minamiyama M, Matsumoto S, Kondo N, Miyazaki Y, IidaM,Tohnai G, Qiang Q, Tanaka F, Yanagawa T, WarabiE,Ishii T, Sobue G
  • Journal Title: J Neurosci Volume: 33 Issue: 18 Pages: 7710-7727
  • DOI: 10.1523/jneurosci.3021-12.2013
  • Peer Reviewed
• [Journal Article] Translational research on disease-modifying therapies for neurodegenerative diseases (2013)
  • Author(s): Katsuno M, Watanabe H, Tanaka F, Sobue G
  • Journal Title: Neurology and Clinical Neuroscience Volume: 1 Issue: 1 Pages: 3-10
  • DOI: 10.1002/ncn3.7
  • Peer Reviewed
• [Journal Article] Genistein, a natural product derived from soybeans, ameliorates polyglutamine-mediated motor neuron disease (2013)
  • Author(s): Qiang Q, Adachi H, Huang Z, Jiang YM, Katsuno M, Minamiyama M, Doi H, Matsumoto S, Kondo N, Miyazaki Y, Iida M, Tohnai G, Sobue G
  • Journal Title: J Neurochem Volume: 126 Issue: 1 Pages: 122-130
  • DOI: 10.1111/jnc.12172
  • Peer Reviewed
• [Journal Article] Heat shock factor-1 influences pathological lesion distribution of polyglutamine-induced neurodegeneration (2013)
  • Author(s): Kondo N, Katsuno M, Adachi H, Minamiyama M, Doi H, Matsumoto S, Miyazaki Y, Iida M, Tohnai G, Nakatsuji H, Ishigaki S, Fujioka Y, Watanabe H, Tanaka F, Nakai A, Sobue G
  • Journal Title: Nat Commun Volume: 4 Issue: 1 Pages: 1405-1405
  • DOI: 10.1038/ncomms2417
  • Peer Reviewed
• [Journal Article] Distinct acoustic features in spinal and bulbar muscular atrophy patients with laryngospasm. (2013)
  • Author(s): Tanaka S, Banno H, Katsuno M, Suzuki K, Suga N, Hashizume A, Mano T, Araki A, Watanabe H, Adachi H, Tatsumi H, Yamamoto M, Sobue G.
  • Journal Title: J Neurol Sci. Volume: 337 Issue: 1-2 Pages: 193-200
  • DOI: 10.1016/j.jns.2013.12.010
  • Peer Reviewed
• [Journal Article] A functional deficiency of TERA/VCP/p97 contributes to impaired DNA repair in multiple polyglutamine diseases. (2013)
  • Author(s): Fujita K, Nakamura Y, Oka T, Ito H, Tamura T, Tagawa K, Sasabe T, Katsuta A, Motoki K, Shiwaku H, Sone M, Yoshida C, Katsuno M, Eishi Y, Murata M, Taylor JP, Wanker EE, Kono K, Tashiro S, Sobue G, La Spada AR, Okazawa H.
  • Journal Title: Nat Commun. Volume: 4 Issue: 1 Pages: 1816-1816
  • DOI: 10.1038/ncomms2828
  • Peer Reviewed
• [Journal Article] Androgen receptor inclusions acquire GRP78/BiP to ameliorate androgen-induced protein misfolding stress in embryonic stem cells. (2013)
  • Author(s): Yang YC, Fu HC, Hsiao BL, Sobue G, Adachi H, Huang FJ, Hsuuw YD, Wei KT, Chang C, Huang KE, Kang HY.
  • Journal Title: Cell Death Dis. Volume: 4 Issue: 4 Pages: e607-e607
  • DOI: 10.1038/cddis.2013.122
  • Peer Reviewed
• [Journal Article] IGF-1 administration ameliorates disease manifestations in a mouse model of spinal and bulbar muscular atrophy (2012)
  • Author(s): Rinaldi C, Bott L, Chen K, Harmison G, Katsuno M, Sobue G, Pennuto M, Fischbeck KH
  • Journal Title: Mol Med Volume: 18 Issue: 9 Pages: 1261-1268
  • DOI: 10.2119/molmed.2012.00271
  • Peer Reviewed
• [Journal Article] Viral delivery of miR-196a ameliorates the SBMA phenotype via the silencing of CELF2 (2012)
  • Author(s): Miyazaki Y, Adachi H, Katsuno M, Minamiyama M, Jiang YM, Huang Z, Doi H, Matsumoto S, Kondo N, Iida M, Tohnai G, Tanaka F, Muramatsu S, Sobue G
  • Journal Title: Nat Med Volume: 18 Issue: 7 Pages: 1136-1141
  • DOI: 10.1038/nm.2791
  • Peer Reviewed
• [Journal Article] Naratriptan mitigates CGRP1-associated motor neuron degeneration caused by expanded polyglutamine (2012)
  • Author(s): Minamiyama M, Katsuno M, Adachi H, Doi H, Kondo N, Iida M, Ishigaki S, Fujioka Y, Matsumoto S, Miyazaki Y, Tanaka F, Kurihara H, Sobue G
  • Journal Title: Nat Med Volume: 18 Issue: 10 Pages: 1531-1538
  • DOI: 10.1038/nm.2932
  • Peer Reviewed
• [Journal Article] Pathogenesis and therapy of spinal and bulbar muscular atrophy (SBMA) (2012)
  • Author(s): Katsuno M, Tanaka F, Adachi H, Banno H, Suzuki K, Watanabe H, Sobue G
  • Journal Title: Prog Neurobiol Volume: 99 Issue: 3 Pages: 246-256
  • DOI: 10.1016/j.pneurobio.2012.05.007
  • Peer Reviewed
• [Journal Article] Current status of treatment of spinal and bulbar muscular atrophy (2012)
  • Author(s): Tanaka F, Katsuno M, Banno H, Suzuki K, Adachi H, Sobue G
  • Journal Title: Neural Plast Volume: 2012 Pages: 369284-369284
  • DOI: 10.1155/2012/369284
  • Peer Reviewed
• [Journal Article] Molecular pathophysiology and disease-Modifying therapies for spinal and bulbar muscular atrophy (2012)
  • Author(s): Katsuno M, Banno H, Suzuki K, Adachi H, Tanaka F, Sobue G
  • Journal Title: Arch Neurol Volume: 69 Issue: 4 Pages: 436-440
  • DOI: 10.1001/archneurol.2011.2308
  • Peer Reviewed
• [Journal Article] Perspectives on molecular targeted therapies and clinical trials for neurodegenerative diseases (2012)
  • Author(s): Katsuno M, Tanaka F, Sobue G
  • Journal Title: J Neurol Neurosurg Psychiatry Volume: 83 Issue: 3 Pages: 329-335
  • DOI: 10.1136/jnnp-2011-301307
  • Peer Reviewed
• [Journal Article] Disruption of axonal transport in motor neuron diseases (2012)
  • Author(s): Ikenaka K, Katsuno M, Kawai K, Ishigaki S, Tanaka F, Sobue G
  • Journal Title: Int J Mol Sci Volume: 13 Pages: 1225-1238
  • Peer Reviewed
• [Journal Article] Longitudinal changes of outcome measures in spinal and bulbar muscular atrophy. (2012)
  • Author(s): Hashizume, A., Katsuno, M., Banno, H., Suzuki, K., Suga, N., Mano, T., Atsuta, N., Oe, H., Watanabe, H., Tanaka, F., Sobue, G.
  • Journal Title: Brain Volume: 35 Issue: 9 Pages: 2838-2848
  • DOI: 10.1093/brain/aws170
  • Peer Reviewed
• [Journal Article] Cross-sectional and longitudinal analysis of an oxidative stress biomarker for spinal and bulbar muscular atrophy. (2012)
  • Author(s): Mano, T., Katsuno, M., Banno, H., Suzuki, K., Suga, N., Hashizume, A., Tanaka, F., Sobue, G.
  • Journal Title: Muscle Nerve Volume: 46 Issue: 5 Pages: 692-697
  • DOI: 10.1002/mus.23413
  • Peer Reviewed
• [Journal Article] Pathogenesis and molecular targeted therapy of spinal and bulbar muscular atrophy (SBMA). (2012)
  • Author(s): Banno, H., Katsuno, M., Suzuki, K., Tanaka, F., Sobue, G.
  • Journal Title: Cell Tissue Res Volume: 349 Issue: 1 Pages: 313-320
  • DOI: 10.1007/s00441-012-1377-9
  • Peer Reviewed
• [Journal Article] Discrimination of spinal and bulbar muscular atrophy from amyotrophic lateral sclerosis using sensory nerve action potentials. (2012)
  • Author(s): Hama T, Hirayama M, Hara T, Nakamura T, Atsuta N, Banno H, Suzuki K, Katsuno M, Tanaka F, Sobue G.
  • Journal Title: Muscle Nerve Volume: 45 Issue: 2 Pages: 169-174
  • DOI: 10.1002/mus.22291
  • Peer Reviewed
• [Journal Article] Oxidative stress induced by glutathione depletion reproduces pathological modifications of TDP-43 linked to TDP-43 proteinopathies. (2012)
  • Author(s): Iguchi Y,Katsuno M,Takagi S.Ishigaki S,Niwa JI,Hasegawa M,Tanaka F,Sobue G
  • Journal Title: Neurobiol Dis Volume: 45 Issue: 3 Pages: 862-870
  • DOI: 10.1016/j.nbd.2011.12.002
  • Peer Reviewed
• [Journal Article] Differential change of clinical outcome measures in spinal and bulbar muscular atrophy: comparison of natural history with placebo-treated group. (2012)
  • Author(s): Hashizume A, Katsuno M, Banno H, Suzuki K, Suga N, Tanaka F, Sobue G.
  • Journal Title: J Neurol. Volume: 259 Issue: 4 Pages: 712-719
  • DOI: 10.1007/s00415-011-6251-2
  • Peer Reviewed
• [Journal Article] Defective axonal transport in motor neuron diseases. (2012)
  • Author(s): Ikenaka K, Katsuno M, Kawai K, Ishigaki S, Tanaka F, Sobue G.
  • Journal Title: Int J Mol Sci. Volume: 13 Issue: 1 Pages: 1225-1238
  • DOI: 10.3390/ijms13011225
  • Peer Reviewed
• [Journal Article] Macroautophagy is regulated by the UPR-mediator CHOP and accentuates the phenotype of SBMA mice (2011)
  • Author(s): Yu Z, Wang AM, Adachi H, Katsuno M, Sobue G, Yue Z, Robins DM, Lieberman AP
  • Journal Title: PLoS Genet Volume: 7 Issue: 10 Pages: e1002321-e1002321
  • DOI: 10.1371/journal.pgen.1002321
  • Peer Reviewed
• [Journal Article] A functional variant in ZNF512B is associated with susceptibility to amyotrophic lateral sclerosis in Japanese. (2011)
  • Author(s): Iida A
  • Journal Title: Hum Mol Genet Volume: 20 Issue: 18 Pages: 3684-3692
  • DOI: 10.1093/hmg/ddr268
  • Peer Reviewed
• [Journal Article] Suppression of Alzheimer's disease-related phenotypes by expression of heat shock protein 70 in mice. (2011)
  • Author(s): Hoshino T, Murao N, Namba T, Takehara M, Adachi H, Katsuno M, Sobue G, Matsushima T, Suzuki T, Mizushima T.
  • Journal Title: J Neurosci. Volume: 31 Issue: 14 Pages: 5225-5234
  • DOI: 10.1523/jneurosci.5478-10.2011
  • Peer Reviewed
• [Journal Article] Dutasteride for spinal and bulbar muscular atrophy : another advance to wards disease-modifying therapy. (2011)
  • Author(s): Banno H, Katsuno M, Suzuki K, Sobue G.
  • Journal Title: Lancet Neurol. Volume: 10 Pages: 113-115
• [Journal Article] Transforming growth factor-β signaling in motor neuron diseases. (2011)
  • Author(s): Katsuno M, Adachi H, Banno H, Suzuki K, Tanaka F, Sobue G.
  • Journal Title: Current Mol Med. Volume: 11 Pages: 48-56
  • Peer Reviewed
• [Journal Article] Disrupted TGF-beta signaling in spinal and bulbar muscular atrophy (2010)
  • Author(s): Katsuno M, Adachi H, Minamiyama M, Waza M, Doi H, Kondo N, Mizoguchi H, Nitta A, Yamada K, Banno H, Suzuki K, Tanaka F, Sobue G
  • Journal Title: J Neurosci Volume: 30 Issue: 16 Pages: 5702-5712
  • DOI: 10.1523/jneurosci.0388-10.2010
  • Peer Reviewed
• [Journal Article] Microarray analysis of gene expression by skeletal muscle of three mouse models of kennedy disease/spinal bulbar muscular atrophy. (2010)
  • Author(s): Mo K, Razak Z, Rao P, Yu Z, Adachi H, Katsuno M, Sobue G, Lieberman AP, Westwood JT, Monks DA.
  • Journal Title: PLoS One Volume: 5
  • Peer Reviewed
• [Journal Article] Clinical features and molecular mechanisms of spinal and bulbar muscular atrophy (SBMA). (2010)
  • Author(s): Katsuno M, Banno H, Suzuki K, Adachi H, Tanaka F, Sobue G.
  • Journal Title: Adv Exp Med Biol. Volume: 685 Pages: 64-74
  • Peer Reviewed
• [Journal Article] Disrupted transforming growth factor-beta signaling in spinal and bulbar muscular atrophy. (2010)
  • Author(s): Katsuno M, Adachi H, Minamiyama M, Waza M, Doi H, Kondo N, Mizoguchi H, Nitta A, Yamada K, Banno H, Suzuki K, Tanaka F, Sobue G
  • Journal Title: J Neurosci Volume: 30 Pages: 5702-5712
  • Peer Reviewed
• [Journal Article] Efficacy and safety of leuprorelin in patients with spinal and bulbar muscular atrophy (JASMITT study) : a multicentre, randomised, double-blind, placebo-controlled trial. (2010)
  • Author(s): Katsuno M, Banno H, Suzuki K, Takeuchi Y, Kawashima M, Yabe I, Sasaki H, Aoki M, Morita M, Nakano I, Kanai K, Ito S, Ishikawa K, Mizusawa H, Yamamoto T, Tsuji S, Hasegawa K, Shimohata T, Nishizawa M, Miyajima H, Kanda F, Watanabe Y, Nakashima K, Tsujino A, Yamashita T, Uchino M, Fujimoto Y, Tanaka F, Sobue Cr ; for the Japan SBMA Interventional Trial for TAP-144-SR (JASMITT) study group.
  • Journal Title: Lancet Neurol. Volume: 9 Pages: 875-884
  • Peer Reviewed
• [Journal Article] The profile of motor unit number estimation (MUNE) in spinal and bulbar muscular atrophy (2010)
  • Author(s): Suzuki K, Katsuno M, Banno H, Takeuchi Y, Kawashima M, Suga N, Hashizume A, Hama T, Uchida K, Yamashita F, Nakamura T, Hirayama M, Tanaka F, Sobue G.
  • Journal Title: J Neurol Neurosurg Psychiatry 81 Pages: 567-571
  • Peer Reviewed
• [Journal Article] Disrupted TGF-beta signaling in spinal and bulbar muscular atrophy (2010)
  • Author(s): Katsuno M, Adachi H, Minamiyama M, Waza M, Doi H, Kondo N, Mizoguchi H, Nitta A, Yamada K, Banno H, Suzuki K, Tanaka F, Sobue G.
  • Journal Title: J Neurosci. 30 Pages: 5702-5712
  • Peer Reviewed
• [Journal Article] Dorfin ameliorates phenotypes in a transgenic mouse model of amyotrophic lateral sclerosis (2010)
  • Author(s): Sone J, Niwa JI, Kawai K, Ishigaki S, Yamada SI, Adachi H, Katsuno M, Tanaka F, Doyu M, Sobue G.
  • Journal Title: J Neurosci Res. 88 Pages: 123-135
  • Peer Reviewed
• [Journal Article] Heat shock proteins in neurodegenerative diseases : Pathogenic roles and therapeutic implications (2009)
  • Author(s): Adachi H, Katsuno M, Waza M, Minamiyama M, Tanaka F, Sobue G.
  • Journal Title: Int J Hyperthermia 25 Pages: 647-654
• [Journal Article] Correlation between pyramidal tract degeneration and widespread white matter involvement in amyotrophic lateral sclerosis : a study with tractography and diffusion-tensor imaging (2009)
  • Author(s): Senda J, Ito M, Watanabe H, Atsuta N, Kawai Y, Katsuno M, Tanaka F, Naganawa S, Fukatsu H, Sobue G.
  • Journal Title: Amyotroph Lateral Scler. 10 Pages: 288-294
  • Peer Reviewed
• [Journal Article] Overexpression of IGF-1 in muscle attenuates disease in a mouse model of spina I and bulbar muscular atrophy (2009)
  • Author(s): Palazzolo I, Stack C, Kong L, Musaro A, Adachi H, Katsuno M, Sobue G, Taylor JP, Sumner CJ, Fischbeck KH, Pennuto M.
  • Journal Title: Neuron 63 Pages: 316-328
  • Peer Reviewed
• [Journal Article] TDP-43 Depletion Induces Neuronal Cell Damage through Dysregulation of Rho Family GTPases (2009)
  • Author(s): Iguchi Y, Katsuno M, Niwa J, Yamada S, Sone J, Waza M, Adachi H, Tanaka F, Nagata K, Arimura N, Watanabe T, Kaibuchi K, Sobue G.
  • Journal Title: J Biol Chem. 284 Pages: 22059-22066
  • Peer Reviewed
• [Presentation] Genistein, a natural product derived from soybeans, ameliorates polyglutamine-mediated motor neuron disease (2013)
  • Author(s): Adachi H, Qiang Q, Hunag Z, Jinag YM, Katsuno M, Kondo N, Miyazaki Y, Iida M, Tohnai G, Nakatsuji H, Sobue G
  • Organizer: Neuroscience 2013
  • Place of Presentation: San Diego, USA
• [Presentation] What can we learn from Kennedy's disease for amyotrophic lateral sclerosis/motor neuron disease (2013)
  • Author(s): Sobue G
  • Organizer: World Congress of Neurology 2013
  • Place of Presentation: Vienna
• [Presentation] Molecular targeted therapy for spinal and bulbar muscular atrophy (2013)
  • Author(s): Sobue G
  • Organizer: Gordon Research Conference CAG Triplet Repeat Disorders
  • Place of Presentation: Waterville Valley, USA
• [Presentation] Deficits in brain-derived neurotrophic factor may underlie motor dysfunction in spinal bulbar muscular atrophy. (2013)
  • Author(s): Hlievski, K., Henley, C.L., Fu, M., Katsuno, M., Adachi, H., Sobue, G., Breedlove, S.M., Jordan, C.L.
  • Organizer: Neuroscience 2013
  • Place of Presentation: San Diego, USA
• [Presentation] Genistein, a natural product derived from soybeans, ameliorates polyglutamine-mediated motor neuron disease. (2013)
  • Author(s): Adachi, H., Qiang, Q., Hunag, Z., Jinag, Y.M., Katsuno, M., Kondo, N., Miyazaki, Y., Iida, M., Tohnai, G., Nakatsuji, H., Sobue, G.
  • Organizer: Neuroscience 2013
  • Place of Presentation: San Diego, USA
• [Presentation] Heat shock factor-1 (Hsf-1) controls pathological lesion distribution of polyglutamine-induced motor neuron disease. (2013)
  • Author(s): Kondo, N., Katsuno, M., Adachi, H., Minamiyama, M., Matsumoto, S., Nakatsuji, H., Miyazaki, Y., Iida, M., Tohnai, G., Sobue, G.
  • Organizer: The 24th International Symposium on ALS/MND
  • Place of Presentation: Milan, Italy
• [Presentation] Molecular targeted therapy for spinal and bulbar muscular atrophy. (2013)
  • Author(s): Sobue G.
  • Organizer: Gordon Research Conference CAG Triplet Repeat Disorders
  • Place of Presentation: Waterville Valley, USA
  • Invited
• [Presentation] What can we learn from Kennedy's disease for amyotrophic lateral sclerosis/motor neuron disease. (2013)
  • Author(s): Sobue G.
  • Organizer: World Congress of Neurology 2013
  • Place of Presentation: Vienna, Austria
  • Invited
• [Presentation] Cell cycle dysregulation in motor neurons of spinal and bulbar muscular atrophy (SBMA) (2012)
  • Author(s): Katsuno M, Adachi H, Kondo N, Minamiyama M, Doi H, Matsumoto S, Miyazaki Y, Iida M, Nakatsuji H, Tanaka F, Sobue G
  • Organizer: 23rd International Symposium on ALS/MND
  • Place of Presentation: Chicago, USA
• [Presentation] p62/SQSTM1 removes toxic mutant androgen receptor in spinal bulbar muscular atrophy model mouse (2012)
  • Author(s): Adachi H, Doi H, Katsuno M, Matsumoto S, Kondo N, Miyazaki Y, Tohnai G, Iida M, Tanaka F, Sobue G
  • Organizer: Neuroscience 2012
  • Place of Presentation: New Orleans, USA
• [Presentation] Viral delivery of miR-196a ameliorates the SBMA phenotype via the silencing of CELF2 (2012)
  • Author(s): Miyazaki Y, Adachi H, Katsuno M, Tanaka F, Muramatsu SI, Sobue G
  • Organizer: Neuroscience 2012
  • Place of Presentation: New Orleans, USA
• [Presentation] Naratriptan ameliorates SBMA pathology by the repression of CGRP1-activated JNK pathway (2012)
  • Author(s): Minamiyama M, Katsuno M, Adachi H, Doi H, Kondo N, Iida M, Ishigaki S, Fujioka Y, Matsumoto S, Miyazaki Y, Tanaka F, Kurihara H, Sobue G
  • Organizer: Neuroscience 2012
  • Place of Presentation: New Orleans, USA
• [Presentation] p62/SQSTM1 removes toxic mutant androgen receptor in spinal bulbar muscular atrophy model mouse. (2012)
  • Author(s): Adachi, H., Doi, H., Katsuno, M., Matsumoto, S., Kondo, N., Miyazaki, Y., Tohnai, G., Iida, M., Tanaka, F., Sobue, G.
  • Organizer: Neuroscience 2012.
  • Place of Presentation: New Orleans, USA
• [Presentation] Viral delivery of miR-196a ameliorates the SBMA phenotype via the silencing of CELF2. (2012)
  • Author(s): Miyazaki, Y., Adachi, H., Katsuno, M., Tanaka, F., Muramatsu, S.I., Sobue, G.
  • Organizer: Neuroscience 2012.
  • Place of Presentation: New Orleans, USA
• [Presentation] Naratriptan ameliorates SBMA pathology by the repression of CGRP1-activated JNK pathway. (2012)
  • Author(s): Minamiyama, M., Katsuno, M., Adachi, H., Doi, H., Kondo, N., Iida, M., Ishigaki, S., Fujioka, Y., Matsumoto, S., Miyazaki, Y., Tanaka, F., Kurihara, H., Sobue, G.
  • Organizer: Neuroscience 2012.
  • Place of Presentation: New Orleans, USA
• [Presentation] Molecular pathogenesis and translational research in spinal and bulbar muscular atrophy (SBMA) (2011)
  • Author(s): Sobue G
  • Organizer: 22th International Symposium on ALS/MND
  • Place of Presentation: Sydney, Australia
• [Presentation] A peony extract enhances protein degradation systems and exerts therapeutic effects in the polyglutamine-mediated motor neuron disease (2011)
  • Author(s): Adachi H, Tohnai G, Katsuno M, Minamiyama M, Doi H, Matsumoto S, Kondo N, Miyazaki Y, Tanaka F, Ohtsuka K, Sobue G
  • Organizer: Neuroscience 2011
  • Place of Presentation: Washington DC, USA
• [Presentation] Heat shock factor-1(Hsf-1) influences distribution of pathogenic androgen receptor aggregations in model mouse of spinal and bulbar muscular atrophy (SBMA) (2011)
  • Author(s): Kondo N, Katsuno M, Adachi H, Minamiyama M, Doi H, Matsumoto S-J, Miyazaki Y, Tanaka F, Sobue G
  • Organizer: Gordon conference on CAG triplet repeat disorders
  • Place of Presentation: Il Ciocco, Italy
• [Presentation] Cell cycle dysregulation in spinal and bulbar muscular atrophy (SBMA) (2011)
  • Author(s): Katsuno M, Adachi H, Kondo N, Minamiyama M, Doi H, Matsumoto S-J, Miyazaki Y, Tanaka F, Sobue G
  • Organizer: Gordon conference on CAG triplet repeat disorders
  • Place of Presentation: Il Ciocco, Italy
• [Presentation] Molecular pathogenesis and translational research in spinal and bulbar muscular atrophy (SBMA). (2011)
  • Author(s): Sobue G.
  • Organizer: 22th International Symposium on ALS/MND
  • Place of Presentation: Sydney, Australia
  • Invited
• [Presentation] TGF-betaからみた神経変性機序. (2011)
  • Author(s): 勝野雅央
  • Organizer: 第52回日本神経学会学術大会シンポジウム「神経変性をどう考えるか？病態理解に至る最近の進歩」
  • Place of Presentation: 名古屋国際会議場(愛知）
  • Invited
• [Presentation] Effect of leuprorelin in patients with spinal and bulbar muscular atrophy (SBMA): a multicenter, randomized, double-blind, placebo-controlled trial. (2011)
  • Author(s): Banno H
  • Organizer: 63rd American Academy of Neurology Annual Meeting
  • Place of Presentation: Honolulu, USA
• [Presentation] Long-term effects of the luteinizing hormone-releasing hormone analogues for spinal and bulbar muscular atrophy. (2011)
  • Author(s): Ota K, Ishikawa K, Suzuki K, Banno H, Katsuno M, Sobue G, Mizusawa H.
  • Organizer: 63rd American Academy of Neurology Annual Meeting
  • Place of Presentation: Honolulu, USA
• [Presentation] Central motor conduction time (CMCT) in patients with spinal and bulbar muscular atrophy (SBMA). (2011)
  • Author(s): Suzuki K, Katsuno M, Banno H, Suga N, Hashizume A, Hara T, Hama T, Nakamura T, Hirayama M, Tanaka F, Sobue G.
  • Organizer: 15th Congress of the European Federation of Neurological Societies
  • Place of Presentation: Budapest, Hungary
• [Presentation] Cell cycle dysregulation in spinal and bulbar muscular atrophy (SBMA). (2011)
  • Author(s): Katsuno M, Adachi H, Kondo N, Minamiyama M, Doi H, Matsumoto S-J, Miyazaki Y, Tanaka F, Sobue G.
  • Organizer: Gordon conference on CAG triplet repeat disorders
  • Place of Presentation: Il Ciocco, Italy,
• [Presentation] Heat shock factor-1 (Hsf-1) influences distribution of pathogenic androgen receptor aggregations in model mouse of spinal and bulbar muscular atrophy (SBMA). (2011)
  • Author(s): Kondo N, Katsuno M, Adachi H, Minamiyama M, Doi H, Matsumoto S-J, Miyazaki Y, Tanaka F, Sobue G.
  • Organizer: Gordon conference on CAG triplet repeat disorders
  • Place of Presentation: Il Ciocco, Italy,
• [Presentation] 球脊髄性筋萎縮症における細胞周期異常. (2011)
  • Author(s): 勝野雅央, 足立弘明, 南山 誠, 土井英樹, 近藤直英, 松本慎二郎, 宮崎 雄, 坂野晴彦, 鈴木啓介, 田中章景, 祖父江 元.
  • Organizer: 第52回日本神経学会学術大会
  • Place of Presentation: 名古屋国際会議場（愛知）
• [Presentation] 球脊髄性筋萎縮症モデルマウスにおける異常蛋白凝集の病理学的分布はHsf-1の影響を受ける. (2011)
  • Author(s): 近藤直英, 勝野雅央, 足立弘明, 南山誠, 土井英樹, 松本慎二郎, 宮崎雄, 飯田円, 田中章景, 祖父江元.
  • Organizer: 第52回日本神経学会学術大会
  • Place of Presentation: 名古屋国際会議場（愛知）
• [Presentation] 球脊髄性筋萎縮症(SBMA)病態関連遺伝子CGRP1の分子機構 (2011)
  • Author(s): 南山 誠, 勝野雅央, 足立弘明, 土井英樹, 近藤直英, 田中章景, 祖父江 元, 栗原裕基.
  • Organizer: 第52回日本神経学会学術大会
  • Place of Presentation: 名古屋国際会議場（愛知）
• [Presentation] Cell cycle dysregulation in spinal and bulbar muscular atrophy (SBMA). (2011)
  • Author(s): Katsuno M, Adachi H, Minamiyama M, Kondo N, Doi H, Matsumoto S, Miyazaki Y, Tanaka F, Sobue G.
  • Organizer: 第34回日本神経科学大会
  • Place of Presentation: パシフィコ横浜（神奈川）
• [Presentation] Molecular pathology of SBMA (Spinal Bulbar Muscular Atrophy) related gene CGRP1. (2011)
  • Author(s): Minamiyama M, Katsuno M, Adachi H, Doi H, Kondo N, Tanaka F, Sobue G, Kurihara H.
  • Organizer: 第34回日本神経科学大会
  • Place of Presentation: パシフィコ横浜（神奈川）
• [Presentation] Heat shock factor-1(HSF-1) influences distribution of pathogenic androgen receptor aggregations in model mouse of spinal and bulbar muscular atrophy (SBMA). (2011)
  • Author(s): Kondo N, Katsuno M, Adachi H, Minamiyama M, Doi H, Matsumoto S, Miyazaki Y, Iida M, Tanaka F, Sobue G.
  • Organizer: 第34回日本神経科学大会
  • Place of Presentation: パシフィコ横浜（神奈川）
• [Presentation] Disrupted TGF-beta signaling in spinal and bulbar muscular atrophy. (2010)
  • Author(s): Katsuno M, Adachi H, Minamiyama M, Doi H, Kondo N, Matsumoto S, Miyazaki Y, Banno H, Suzuki K, Tanaka F, Sobue G.
  • Organizer: 21st International Symposium on ALS/MND.
  • Place of Presentation: Orlando, USA
  • Year and Date: 2010-12-12
• [Presentation] A peony extract alleviates polyglutamine-mediated motor neuron disease (2010)
  • Author(s): Adachi H, Tohnai G, Katsuno M, Minamiyama M, Doi H, Matsumoto S, Kondo N, Miyazaki Y, Tanaka F, Sobue G.
  • Organizer: Neuroscience 2010
  • Place of Presentation: San Diego, USA
  • Year and Date: 2010-11-16
• [Presentation] Oxidative stress induces TDP-43 modification, recapitulating its pathological feature. (2010)
  • Author(s): Iguchi Y, Katsuno M, Takagi S, Sobue G.
  • Organizer: Neuroscience 2010
  • Place of Presentation: San Diego, USA
  • Year and Date: 2010-11-15
• [Presentation] 球脊髄性筋萎縮症に対する臨床治験の取組. (2010)
  • Author(s): 勝野雅央
  • Organizer: 平成22年度厚生労働科学研究費補助金(難治性疾患克服研究事業)「今後の難病対策に関する研究」シンポジウム
  • Place of Presentation: 東京
  • Year and Date: 2010-08-28
• [Presentation] 神経変性疾患の病態に基づく治療への展望 (2010)
  • Author(s): 祖父江元
  • Organizer: 平成22年度「包括脳ネットワーク」夏のワークショップ病態脳科学関連ワークショップ「脳疾患研究の新しい潮流」
  • Place of Presentation: 東京
  • Year and Date: 2010-07-28
• [Presentation] Molecular targeted therapy for Spinal and Bulbar Muscular Atrophy(SBMA) (2010)
  • Author(s): 祖父江元
  • Organizer: 第51回日本神経学会総会シンポジウム「Academia-based development of disease-modifying therapy (アカデミア発の創薬・治療研究)
  • Place of Presentation: 東京
  • Year and Date: 2010-05-21
• [Presentation] 球脊髄性筋萎縮症に対する分子標的治療法の開発 (2010)
  • Author(s): 祖父江元
  • Organizer: 第51回日本神経病理学会総会学術研究会シンポジウム
  • Place of Presentation: 東京
  • Year and Date: 2010-04-23
• [Presentation] 神経変性疾患の分子標的治療への展開:球脊髄性筋萎縮症について (2010)
  • Author(s): 祖父江元
  • Organizer: 第47回日本臨床分子医学会学術集会特別講演
  • Place of Presentation: 東京
  • Year and Date: 2010-04-10
• [Presentation] TDP-43 depletion induces neuronal cell damage through dysregulation of Rho family GTPases (2009)
  • Author(s): Iguchi Y, Katsuno M, Niwa J, Yamada S, Sone J, Waza M, Adachi H, Tanaka F, Nagata K, Arimura N, Watanabe T, Kaibuchi K, Sobue G.
  • Organizer: Neuroscience 2009
  • Place of Presentation: Chicago, USA
  • Year and Date: 2009-10-17
• [Presentation] 球脊髄性筋萎縮症に対する分子治療 (2009)
  • Author(s): 勝野雅央
  • Organizer: 第50回日本神経学会総会シンポジウム
  • Place of Presentation: 仙台
  • Year and Date: 2009-05-22
• [Presentation] Phase 2 trial of leuprorelin in patients with spinal and bulbar muscular atrophy (2009)
  • Author(s): Banno H, Katsuno M, Suzuki K, Takeuchi Y, Kawashima M, Suga N, Takamori M, Ito M, Nakamura T, Matsuo K, Yamada S, Oki Y, Adachi H, Minamiyama M, Waza M, Atsuta N, Watanabe H, Fujimoto Y, Nakashima T, Tanaka F, Doyu M, Sobue G.
  • Organizer: 61th American Academy of Neurology Annual Meeting
  • Place of Presentation: Seattle, USA
  • Year and Date: 2009-04-26
• [Presentation] 神経変性疾患の病態抑止治療への展望
  • Author(s): 祖父江 元
  • Organizer: 第46回日本内科学会中国支部生涯教育講演会
  • Place of Presentation: 出雲医師会館（出雲）
  • Invited
• [Presentation] 神経疾患のdisease modifying therapyへの展開
  • Author(s): 祖父江 元
  • Organizer: 包括型脳科学研究推進支援ネットワーク 夏のワークショップ 脳科学研究戦略推進プログラム・包括脳合同企画
  • Place of Presentation: 仙台国際センター（仙台）
  • Invited
• [Presentation] 神経変性疾患の病体抑止治療を目指して
  • Author(s): 祖父江 元
  • Organizer: 第35回日本神経科学大会特別講演
  • Place of Presentation: 名古屋国際会議場（名古屋）
  • Invited
• [Presentation] 神経変性疾患のdisease-modifying therapyへの展望
  • Author(s): 祖父江 元
  • Organizer: フォーラム2012衛生薬学・環境トキシコロジー 特別講演
  • Place of Presentation: 名古屋観光ホテル（名古屋）
  • Invited
• [Presentation] 神経変性疾患のトランスレーショナルリサーチ
  • Author(s): 勝野雅央, 坂野晴彦, 鈴木啓介, 橋詰 淳, 足立弘明, 田中章景, 祖父江 元.
  • Organizer: 第109回日本内科学会総会・講演会シンポジウム「わが国のトランスレーショナルリサーチとEBM」
  • Place of Presentation: みやこめっせ（京都）
  • Invited
• [Presentation] 運動ニューロン病研究の進歩と治療への展望
  • Author(s): 祖父江 元
  • Organizer: 第109回日本内科学会講演会
  • Place of Presentation: みやこめっせ（京都）
  • Invited
• [Presentation] Endpoints and biomarkers in clinical trials of neurological disorders.
  • Author(s): Katsuno, M.
  • Organizer: The 3rd International Symposium of Early Stage Clinical Trial.
  • Place of Presentation: 県民共済みらいホール（横浜）
  • Invited
• [Presentation] Neuropathy associated with primary systemic vasculitides.
  • Author(s): Sobue G.
  • Organizer: The Asian Pacific Meeting of Vasculitis and ANCA Workshop 2012
  • Place of Presentation: Tokyo, Japan
  • Invited
• [Presentation] Atypical parkinsonism focusing on natural history of MSA.
  • Author(s): Sobue G.
  • Organizer: 20th World Congress of Neurology
  • Place of Presentation: Marrakesh, Morocco
  • Invited
• [Presentation] Sporadic ALS: Search for pathogenesis and therapeutic approach.
  • Author(s): Sobue G.
  • Organizer: 20th World Congress of Neurology
  • Place of Presentation: Marrakesh, Morocco
  • Invited
• [Presentation] Elucidation of neuronal death signaling pathways and development of disease-modifying therapies for Kennedy’s disease.
  • Author(s): Katsuno, M.
  • Organizer: Kennedy’s Disease Association 2011 Annual Conference and Education Symposium
  • Place of Presentation: Bowie, USA
  • Invited
• [Presentation] ALSの神経病理とオミックス
  • Author(s): 祖父江 元
  • Organizer: 第52回日本神経病理学会総会学術研究会シンポジウム「オミックスサイエンスの展開と神経病理学」
  • Place of Presentation: 京都テルサ(京都）
  • Invited
• [Patent(Industrial Property Rights)] 球脊髄性筋萎縮症治療薬 (2009)
  • Inventor(s): 祖父江元, 勝野雅央, 南山誠
  • Industrial Property Rights Holder: 名古屋大学
  • Industrial Property Number: 2009-277101
  • Filing Date: 2009-12-05