| Budget Amount *help |
¥4,940,000 (Direct Cost: ¥3,800,000、Indirect Cost: ¥1,140,000)
Fiscal Year 2015: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2014: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2013: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
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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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