| Project/Area Number |
15K15391
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| Research Category |
Grant-in-Aid for Challenging Exploratory Research
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| Allocation Type | Multi-year Fund |
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| Research Field |
Pediatrics
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| Research Institution | Tottori University |
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Principal Investigator |
Nanba Eiji 鳥取大学, 生命機能研究支援センター, 教授 (40237631)
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| Co-Investigator(Kenkyū-buntansha) |
檜垣 克美 鳥取大学, 生命機能研究支援センター, 准教授 (90294321)
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| Project Period (FY) |
2015-04-01 – 2017-03-31
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| Project Status |
Completed (Fiscal Year 2016)
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| Budget Amount *help |
¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2016: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2015: ¥2,210,000 (Direct Cost: ¥1,700,000、Indirect Cost: ¥510,000)
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| Keywords | シャペロン / ライソゾーム病 / ファブリー病 / ゴーシェ病 / 自己不活型 / シャペロン療法 / 先天代謝異常症 / 変異蛋白質 / 構造異常 / ライソゾーム酵素 / 変異酵素 |
|---|
| Outline of Final Research Achievements |
We developed "self-inactivating novel chaperone" which undergoes structural change under acidic condition in lysosome and dissociates from target enzyme protein by conversion to inactive form. Based on the deoxynojirimycin (DGJ) compound, several candidate compounds were synthesized by adding an orthoester group. The structure of compounds changed by pH change. We studied the effect of these compounds for Fabry and Gaucher diseases using in vitro and cultured cells. As a result, we identified the compounds as original concept. The compounds did not show enzyme inhibition even when used at high concentrations.
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