| Project/Area Number |
15K15406
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| Research Category |
Grant-in-Aid for Challenging Exploratory Research
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| Allocation Type | Multi-year Fund |
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| Research Field |
Embryonic/Neonatal medicine
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| Research Institution | National Center for Child Health and Development |
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Principal Investigator |
OHKURA TAKASHI 国立研究開発法人国立成育医療研究センター, 細胞医療研究部, (非)研究員 (50183223)
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| Co-Investigator(Kenkyū-buntansha) |
梅澤 明弘 国立研究開発法人国立成育医療研究センター, 再生医療センター, 副所長/再生医療センター長 (70213486)
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| Project Period (FY) |
2015-04-01 – 2018-03-31
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| Project Status |
Completed (Fiscal Year 2017)
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| Budget Amount *help |
¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2017: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2016: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2015: ¥780,000 (Direct Cost: ¥600,000、Indirect Cost: ¥180,000)
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| Keywords | 先天性異常 / ライソゾーム病 / 成育医療 / 糖鎖 / I-Cell病 / 細胞移植 / 酵素補充 / 免疫不全動物 / 先天異常学 |
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| Outline of Final Research Achievements |
In this study, we attempted to analyze pathology and develop stem cell transplantation therapy mainly on multienzyme deficiency of enzyme without treatment, among lysosomal diseases called I - cell disease (ICD). IPS cells were prepared from human ICD patient fibroblasts and differentiated into various organ donor cells to clarify the structure of a characteristic metabolic disorder (structural carbohydrate) around organs. As a result, lysosomal enzyme requirement for each organ was clarified, cells having optimal degradative enzyme secretion properties were transplanted into model mice, and the therapeutic effect and comprehensive safety were analyzed and evaluated
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