| Project/Area Number |
24591526
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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 | Jikei University School of Medicine |
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Principal Investigator |
OHASHI TOYA 東京慈恵会医科大学, 医学部, 教授 (60160595)
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| Co-Investigator(Kenkyū-buntansha) |
SHIMADA Yohta 東京慈恵会医科大学, 医学部, 助教 (20560824)
HIGUCHI Takashi 東京慈恵会医科大学, 医学部, 助教 (30595327)
KOBAYASHI Hiroshi 東京慈恵会医科大学, 医学部, 准教授 (90266619)
衞藤 義勝 東京慈恵会医科大学, 医学部, 名誉教授 (50056909)
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| Project Period (FY) |
2012-04-01 – 2016-03-31
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| Project Status |
Completed (Fiscal Year 2015)
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| Budget Amount *help |
¥5,200,000 (Direct Cost: ¥4,000,000、Indirect Cost: ¥1,200,000)
Fiscal Year 2014: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2013: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2012: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
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| Keywords | ライソゾーム病蓄積症 / ファブリー病 / ポンぺ病 / 酵素補充療法 / 抗体 / 免疫寛容 / ライソゾーム病 / 抗CD3抗体 / 経口投与 / ポンペ病 / 免疫寛容療法 |
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| Outline of Final Research Achievements |
Lysosomal storage diseases are characterized by genetically deficient activity of lysosomal enzyme, resulting in intracellular accumulation of various compounds. This causes various clinical symptoms. So far, about 40 lysosomal storage disease are known. As a treatment, enzyme replacement therapy is approved for 7 lysosomal storage diseases. However, development of antibody against infused enzyme negatively impact on therapeutic efficacy. From our study, antibody not only inhibit the enzyme activity but also cellular uptake of enzyme. Oral administration of enzyme prevent the antibody formation in enzyme replacement therapy for lysosomal storage diseases.
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