Developing new therapeutic drugs that inhibit oligomer formation of polyglutamine disease proteins based on the hypothesis of oligomer toxicity in polyglutamine diseases
| Project/Area Number |
25461272
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Research Field |
Neurology
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| Research Institution | Niigata University |
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Principal Investigator |
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| Co-Investigator(Kenkyū-buntansha) |
Onodera Osamu 新潟大学, 脳研究所, 教授 (20303167)
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| Project Period (FY) |
2013-04-01 – 2016-03-31
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| Project Status |
Completed (Fiscal Year 2015)
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| Budget Amount *help |
¥4,940,000 (Direct Cost: ¥3,800,000、Indirect Cost: ¥1,140,000)
Fiscal Year 2015: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2014: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2013: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
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| Keywords | 脊髄小脳変性症 / ポリグルタミン病 / 治療 / 重合体 / 臨床評価法 / 小脳性運動失調 / 新規治療法 / 評価法 / 重合体形成 / SCD / ataxia / polyglutamine disease / therapy / oligomer |
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| Outline of Final Research Achievements |
We developed new therapeutic drugs that inhibit oligomer formation of polyglutamine disease proteins based on the hypothesis of oligomer toxicity in polyglutamine diseases. Using a disease nematode model, we found a small compound that inhibit oligomer formation of disease proteins and ameliorate motor function of the nematode model. We also developed a novel system using iPad for the quantitative assessment of cerebellar ataxia. In addition, we started a clinical trial to investigate the safety and efficiency of varenicline (Champix), a prescription medication used to treat nicotine addiction, in the treatment of spinocerebellar degenerations.
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Report
(4 results)
Research Products
(16 results)
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[Journal Article] A mutation of COX6A1 causes a recessive axonal or mixed form of Charcot-Marie-Tooth disease.2014
Author(s)
Tamiya G, Makino S, Hayashi M, Abe A, Numakura C, Ueki M, Tanaka A, Ito C, Toshimori K, Ogawa N, Terashima T, Maegawa H, Yanagisawa D, Tooyama I, Tada M, Onodera O, Hayasaka K.
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Journal Title
Am J Hum Genet
Volume: 95(3)
Pages: 294-300
Related Report
Peer Reviewed / Open Access
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[Journal Article] Progressive myoclonus epilepsy: extraneuronal brown pigment deposition and system neurodegeneration in the brains of Japanese patients with novel SCARB2 mutations2013
Author(s)
Fu YJ, Aida I, Tada M, Tada M, Toyoshima Y, Takeda S, Nakajima T, Naito H, Nishizawa M, Onodera O, Kakita A, and Takahashi H
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Journal Title
Neuropathol Appl Neurobiol
Volume: doi:10.1111/nan.12057.
Pages: 12057-12057
Related Report
Peer Reviewed
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