| Project/Area Number |
15590864
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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 |
Kidney internal medicine
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| Research Institution | Nippon Medical School |
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Principal Investigator |
UTSUMI Kouichi Nippon Medical School, Second Department of Internal Medicine, Staff Fellow, 医学部, 助手 (60291150)
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| Co-Investigator(Kenkyū-buntansha) |
IINO Yasuhiko Nippon Medical School, Second Department of Internal Medicine, Professor, 医学部, 教授 (60134706)
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| Project Period (FY) |
2003 – 2004
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| Project Status |
Completed (Fiscal Year 2004)
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| Budget Amount *help |
¥2,800,000 (Direct Cost: ¥2,800,000)
Fiscal Year 2004: ¥1,200,000 (Direct Cost: ¥1,200,000)
Fiscal Year 2003: ¥1,600,000 (Direct Cost: ¥1,600,000)
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| Keywords | Fabry disease / Enzyme replacement therapy / α-Galactosidase / vitro nectine receptor / Maltese cross / Globotriaosylceramide |
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| Research Abstract |
Fabry disease is an X-linked lysosomal storage disease resulting from deficient activity of the enzyme α-galacotsidase A. Accumulation of glycosphingolipids, especially globotriaosylceramide, leads to renal damage in Fabry disease. In patients with Fabry disease, the urinary sediment contains excreted glycosphingolipids. With enzyme replacement therapy for Fabry disease now currently available, we examined whether the urinary sediment could be used to noninvasively monitor effectiveness of enzyme replacement therapy. Four male patients with hemizygous classical Fabry disease received recombinant α-galactsidase A biweekly, and urinary sediments were assessed at 3-month intervals. The morphologic and immunohistochemical changes in urinary sediment at 6 and 18 months suggested that accumulations of glycosphingolipids in renal tissues were cleared by enzyme replacement. Examination of urinary sediments could serve as noninvasive monitoring of the effect of therapy in patients with Fabry disease.
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