| Budget Amount *help |
¥4,680,000 (Direct Cost: ¥3,600,000、Indirect Cost: ¥1,080,000)
Fiscal Year 2017: ¥910,000 (Direct Cost: ¥700,000、Indirect Cost: ¥210,000)
Fiscal Year 2016: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2015: ¥2,340,000 (Direct Cost: ¥1,800,000、Indirect Cost: ¥540,000)
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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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