2017 Fiscal Year Final Research Report
Development of molecular therapy for polyglutamine diseases by reverse structure-based drug design
| Project/Area Number |
26713030
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| Research Category |
Grant-in-Aid for Young Scientists (A)
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| Allocation Type | Partial Multi-year Fund |
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| Research Field |
Neurology
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| Research Institution | Osaka University (2016-2017)
Kyoto University (2014-2015) |
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Principal Investigator |
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| Project Period (FY) |
2014-04-01 – 2018-03-31
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| Keywords | ポリグルタミン病 / 分子シャペロン / エクソソーム / 脳内送達 |
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| Outline of Final Research Achievements |
Neurodegenerative diseases, including Alzheimer's disease and Parkinson's disease, are brain diseases with no effective therapies established to date. In this study, we aimed to develop therapy for polyglutamine diseases, one of the neurodegenerative diseases, and obtained three results. (1) We performed detailed analysis on the binding mode of disease-associated polyglutamine proteins with the aggregation inhibitor. (2) We found a novel proteostasis machinery that molecular chaperones are secreted from cells and transmitted to the other cells via exosomes, one of the extracellular vesicles, and suppress neurodegeneration in a non-cell autonomous manner.(3) We successfully developed peptide carriers that can pass through the blood-brain barrier, which can be utilized as brain delivery carriers for drugs. These results provide important knowledge for drug development for polyglutamine diseases and neurodegenerative diseases.
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| Free Research Field |
分子細胞生物学
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