2015 Fiscal Year Final Research Report
New therapeutic approach using functional copper complexes to treat Menkes disase.
| Project/Area Number |
25461534
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | Tohoku University |
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Principal Investigator |
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| Co-Investigator(Kenkyū-buntansha) |
KODAMA Hiroko 帝京大学, 医学部, 非常勤講師 (00093386)
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| Project Period (FY) |
2013-04-01 – 2016-03-31
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| Keywords | Menkes病 / macular mouse |
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| Outline of Final Research Achievements |
Menkes disease is a severe X-chromosome-linked disorder caused by mutations in a copper transporter, ATP7A. We investigated the oral copper supplementation with Cu-gtsm, a lipophilic copper complex, in male hemizygous macular mice, an animal model of Menkes disease. Orally administered Cu-gtsm rescued affected mice with an increase of cerebral copper concentration and activities of cerebral Cu-dependent enzymes including cytochrome oxidase and dopamine-β-hydroxylase. Serum ceruloplasmin activities were also recovered. However, rotarod test performance was still impaired. This study indicates that Cu-gtsm potentially have clinical utility as an oral medication in the treatment of Menkes disease.
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| Free Research Field |
小児神経学
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