2015 Fiscal Year Final Research Report
Establishment of a molecular therapy for the polyglutamine diseases targeting toxic oligomers
| Project/Area Number |
24591287
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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 | Tokyo Medical University (2013-2015)
独立行政法人国立精神・神経医療研究センター (2012) |
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Principal Investigator |
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| Project Period (FY) |
2012-04-01 – 2016-03-31
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| Keywords | 神経変性疾患 / ポリグルタミン病 / 治療薬開発 / オリゴマー |
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| Outline of Final Research Achievements |
Recent studies have demonstrated that in many neurodegenerative diseases, including Alzheimer disease and the polyglutamine (polyQ) diseases, neurodegeneration results from the actions of a toxic oligomeric species of the mutant protein. In this study, we analyzed the effects of QAI1, a small chemical compound inhibitor of polyQ protein oligomers that we previously identified. We analyzed the pharmacokinetics of QAI1 in mice, as well as the therapeutic effects of QAI1 on the neurological phenotypes and histological phenotypes of two mouse models of the polyQ diseases.
We found that QAI1 effectively enters the brain upon its oral administration in mice. Furthermore, oral administration of QAI1 was shown to alleviate the neurological phenotypes and histological phenotypes of 2 mouse models of the polyQ diseases. Our results demonstrated the potential of QAI1 as a therapeutic molecule for the polyQ diseases.
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| Free Research Field |
神経変性疾患
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