2010 Fiscal Year Final Research Report
Molecular therapy for the polyglutamine diseases targeting the toxic β-sheet conformers and oligomers
| Project/Area Number |
20390245
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Neurology
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| Research Institution | National Center of Neurology and Psychiatry |
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Principal Investigator |
NAGAI Yoshitaka National Center of Neurology and Psychiatry, 神経研究所疾病研究第四部, 室長 (60335354)
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| Co-Investigator(Kenkyū-buntansha) |
POPIEL Helena Akiko (独)国立精神・神経医療研究センター, 神経研究所疾病研究第四部, 流動研究員 (40467593)
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| Project Period (FY) |
2008 – 2010
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| Keywords | 神経変性疾患 / ポリグルタミン病 / βシート / オリゴマー / QBP1 / 分子シャペロン / 治療薬 / スクリーニング |
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| Research Abstract |
Toward developing a therapy for the polyglutamine (polyQ) neurodegenerative diseases targeting β-sheet conformers and oligomers of the expanded polyQ protein,1)we showed the modest therapeutic effects of PTD-QBP1 on a mouse model of the polyQ diseases, and determined essential residues within QBP1 for its activity.2)We identified 9 polyQ aggregation inhibitors which exhibit therapeutic effects on a Drosophila model of the polyQ diseases.3)We demonstrated the therapeutic effects of 17-AAG, a chaperone-inducing compound, on polyQ disease Drosophila models.
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[Journal Article] Genetic and clinical analysis in a Chinese parkinsonism-predominant spinocerebellar ataxia type 2 family2011
Author(s)
Sun H, Satake W, Zhang C, Nagai Y, Tian Y, Fu S, Yu J, Qian Y, Qian Y, Chu J, Toda T
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Journal Title
J Hum Genet 56(4)
Pages: 330-334
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[Journal Article] Genetic Testing for Huntington's Disease in Parkinsonism2010
Author(s)
Rahman MS, Nagai Y, Popiel HA, Fujikake N, Okamoto Y, Ahmed MU, Islam MA, Islam MT, Ahmed S, Rahman KM, Uddin MJ, Dey SK, Ahmed Q, Hossain MA, Jahan N, Toda T
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Journal Title
Mymensingh Med J 19(4)
Pages: 510-514
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[Journal Article] Novel gene therapy for polyglutamine diseases to degrade selectively the pathogenic protein2010
Author(s)
Bauer PO, Goswami A, Wong HK, Okuno M, Kurosawa M, Yamada M, Miyazaki H, Matsumoto G, Kino Y, Nagai Y, Nukina N
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Journal Title
Nat Biotechnol 28(3)
Pages: 256-263
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[Journal Article] Residual laminin-binding activity and enhanced dystroglycan glycosylation by LARGE in novel model mice to dystroglycanopathy2009
Author(s)
Kanagawa M, Nishimoto A, Chiyonobu T, Takeda S, Miyagoe-Suzuki Y, Wang F, Fujikake N, Taniguchi M, Lu Z, Tachikawa M, Nagai Y, Tashiro F, Miyazaki J, Tajima Y, Takeda S, Endo T, Kobayashi K, Campbell KP, Toda T
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Journal Title
Hum Mol Genet 18(4)
Pages: 621-631
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