2015 Fiscal Year Final Research Report
Establishment of iPS cells from spinal and bulbar muscular atrophy and the research of polyglutamine diseases
| Project/Area Number |
25860722
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| Research Category |
Grant-in-Aid for Young Scientists (B)
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| Allocation Type | Multi-year Fund |
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| Research Field |
Neurology
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| Research Institution | Keio University |
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Principal Investigator |
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| Project Period (FY) |
2013-04-01 – 2016-03-31
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| Keywords | ポリグルタミン病 / 球脊髄性筋萎縮症 / 神経変性疾患 / 運動ニューロン疾患 / アンドロゲン受容体 / 17-AAG / iPS細胞 |
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| Outline of Final Research Achievements |
We established spinal and bulbar muscular atrophy (SBMA)-derived iPS cells (iPSCs) and confirmed motor neuron differentiation. Aggregation of androgen receptor in SBMA-iPSC-derived neurons is enhanced by dihydrotestosterone. And one of the candidate drugs, 17-AAG promoted to degrade AR aggregation. These findings show iPSCs technology makes us be able to recapitulate disease-specific biochemical features and demonstrate the potential for identification and validation of candidate drugs. We also established iPSCs from other polyglutamine diseases (Machado-Joseph disease, Dentatorubral-pallidoluysian atrophy) and neurodegenerative diseases (amyotrophic lateral sclerosis, Parkinson's disease). We are going to advance the polyglutamine disease researches by using the established iPSC lines.
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| Free Research Field |
神経変性疾患
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