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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

Research Project

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Project/Area Number 15K09606
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeMulti-year Fund
Section一般
Research Field Pediatrics
Research InstitutionMeiji Pharmaceutical University

Principal Investigator

SAKURABA HITOSHI  明治薬科大学, 薬学部, 教授 (60114493)

Co-Investigator(Renkei-kenkyūsha) TSUKIMURA Takahiro  明治薬科大学, 薬学部, 助教 (50632783)
Project Period (FY) 2015-04-01 – 2018-03-31
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
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.

Free Research Field

医歯薬学

URL: 

Published: 2019-03-29  

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