Establishment of iPS cells from spinal and bulbar muscular atrophy and the research of polyglutamine diseases
| Project/Area Number |
25860722
|
|---|
| Research Category |
Grant-in-Aid for Young Scientists (B)
|
| Allocation Type | Multi-year Fund |
|---|
| Research Field |
Neurology
|
|---|
| Research Institution | Keio University |
|---|
Principal Investigator |
|
|---|
| Project Period (FY) |
2013-04-01 – 2016-03-31
|
| Project Status |
Completed (Fiscal Year 2015)
|
| Budget Amount *help |
¥4,030,000 (Direct Cost: ¥3,100,000、Indirect Cost: ¥930,000)
Fiscal Year 2015: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2014: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2013: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
|
|---|
| 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 / 筋萎縮性側索硬化症 / パーキンソン病 / トリプレットリピート病 / 凝集体 / dihydroteststerone |
|---|
| 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.
|
Report
(4 results)
Research Products
(3 results)
