Development of novel small molecules which rescue the trafficking of mutated ABCD1 responsible for adrenoleukodystrophy
| Project/Area Number |
25893038
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| Research Category |
Grant-in-Aid for Research Activity Start-up
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| Allocation Type | Single-year Grants |
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| Research Field |
Drug development chemistry
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| Research Institution | National Institute of Health Sciences |
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Principal Investigator |
MISAWA Takashi 国立医薬品食品衛生研究所, 有機化学部, 研究員 (40709820)
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| Project Period (FY) |
2013-08-30 – 2015-03-31
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| Project Status |
Completed (Fiscal Year 2014)
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| Budget Amount *help |
¥2,730,000 (Direct Cost: ¥2,100,000、Indirect Cost: ¥630,000)
Fiscal Year 2014: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2013: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
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| Keywords | 副腎白質ジストロフィー / 超長鎖脂肪酸 / ABCD1 / ファーマコロジカルシャペロン |
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| Outline of Final Research Achievements |
Adrenoleukodystophy (ALD) is one of peroxisomal diseases which is caused by disorder of peroxisomal fatty acid beta oxidation. The novel agents for the treatment of ALD are urgently needed. many studies have been performed in ALD patients, and the cause of the disease was shown to be a hereditary defect in the expression of ABCD1 (ATP-binding cassette protein D1).In this study, We performed the structural development of peroxisome proliferator-activated receptor ligands for improvement of the expression level and mature of mutated ABCD1, and tried to construct the assay systems for evaluation of effects of the synthesized compounds on mutated ABCD1.
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Report
(3 results)
Research Products
(3 results)
