2017 Fiscal Year Final Research Report
Development of new enzyme replacement therapy for Fabry disease with a high-functioning enzyme expected to escape from harmful immune reaction
| Project/Area Number |
15K09606
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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 | Meiji Pharmaceutical University |
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Principal Investigator |
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| Co-Investigator(Renkei-kenkyūsha) |
TSUKIMURA Takahiro 明治薬科大学, 薬学部, 助教 (50632783)
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| Project Period (FY) |
2015-04-01 – 2018-03-31
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| Keywords | ファブリー病 / 酵素補充療法 / 改変酵素 / α-ガラクトシダーゼ A / α-N-アセチルガラクトサミニダーゼ |
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| Outline of Final Research Achievements |
A modified α-N-acetylgalactosaminidase with α-galactosidase A- like substrate specificity was produced in Chinese hamster ovary cells. The enzyme was incorporated into cultured Fabry cells and stably functioned. Recurrent administration of the enzyme not only improved the pathological changes in the liver, kidneys and heart of a young Fabry mouse but also prevented accumulation of glycolipids in their organs and tissues. Furthermore, it did not induce any harmful immune reaction during the treatment. It is highly promising as a new and safe enzyme for enzyme replacement therapy for Fabry disease.
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| Free Research Field |
医歯薬学
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