2010 Fiscal Year Final Research Report
Preparation and application of mouse model for the study on chaperone therapy for Fabry disease
| Project/Area Number |
20590300
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Pathological medical chemistry
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| Research Institution | Obihiro University of Agriculture and Veterinary Medicine |
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Principal Investigator |
ISHII Satoshi Obihiro University of Agriculture and Veterinary Medicine, 医学部, 客員研究員 (00222935)
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| Co-Investigator(Kenkyū-buntansha) |
MATSUDA Junichiro (独)医薬基盤研究所, 生物資源研究部, 研究リーダー (60181731)
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| Project Period (FY) |
2008 – 2010
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| Keywords | ファブリー病 / シャペロン療法 / 応用動物 |
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| Research Abstract |
The purpose of our study is to establish a mouse model for the chaperone therapy for Fabry disease. Fabry disease is an inherited disease caused by the deficient activity of α-galactosidase A and resulted to accumulation of glycosphingolipid, predominantly globotriaosylceramide (Gb3). The mouse model established previously was good for the determination of enzyme activity but not for the accumulation of Gb3 on the effect of drug candidates. In our present study, we prepared the transgenic mouse expressing human Gb3 synthase gene, and we succeeded a mouse model in which we can determine the effect of compounds on both enzyme activity and Gb3 content.
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