Devlopment of a novel allosteric chaperone therapy for genetic diseases.
| Project/Area Number |
25293230
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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| Allocation Type | Partial Multi-year Fund |
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| Section | 一般 |
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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) |
KAWATA YASUSHI 国立大学法人鳥取大学, 工学研究科, 教授 (40177697)
HIGAKI KATSUMI 国立大学法人鳥取大学, 生命機能研究支援センター, 准教授 (90294321)
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| Project Period (FY) |
2013-04-01 – 2016-03-31
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| Project Status |
Completed (Fiscal Year 2015)
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| Budget Amount *help |
¥18,460,000 (Direct Cost: ¥14,200,000、Indirect Cost: ¥4,260,000)
Fiscal Year 2015: ¥5,330,000 (Direct Cost: ¥4,100,000、Indirect Cost: ¥1,230,000)
Fiscal Year 2014: ¥6,240,000 (Direct Cost: ¥4,800,000、Indirect Cost: ¥1,440,000)
Fiscal Year 2013: ¥6,890,000 (Direct Cost: ¥5,300,000、Indirect Cost: ¥1,590,000)
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| Keywords | 先天代謝異常症 / ライソゾーム病 / アロステリックシャペロン / 脳疾患 / 治療法開発 / シャペロン / 遺伝病 / 治療 / シャペロン化合物 / アロステリック効果 / 蛋白質立体構造 / 神経変性疾患 / 脳変成疾患 / 脂質代謝異常症 / 蛋白質構造 |
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| Outline of Final Research Achievements |
In this study, we have explored to develop non-inhibitory chaperone compounds for human beta-galactosidase deficiency. Screening of the compound library found two candidates, which showed stabilization activity to beta-galactosidase enzyme in vitro without substrate competitive inhibition activity. We also identified a new chaperone candidate, conduramine-F4 based on a lead compound, NOEV. This compound showed a decreased inhibitory activity and increased enzyme enhancing activity against human beta-galactosidase in vitro, indidated as a new chaperone candidate for this disease.
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Report
(4 results)
Research Products
(15 results)
