2017 Fiscal Year Final Research Report
Research on elucidation of pathology and drug discovery in peroxisomal diseases using stem cells and diseased model organisms
| Project/Area Number |
15K15389
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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 |
Pediatrics
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| Research Institution | Gifu University |
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Principal Investigator |
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| Co-Investigator(Renkei-kenkyūsha) |
TAKASHIMA Shigeo 岐阜大学, 生命科学総合研究支援センター, 助教 (50537610)
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| Project Period (FY) |
2015-04-01 – 2018-03-31
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| Keywords | ペルオキシソーム病 / モデルフィッシュ / ゲノム編集 / Zellweger症候群 / 脂肪肝 / 極長鎖脂肪酸 |
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| Outline of Final Research Achievements |
As a result of 3 years of research, we succeeded in creating two types of Zellweger syndrome model fish, severe type and mild type, by genome editing technique. In severe form, there were abnormal peroxisomal metabolic functions and phenotypes reflecting the pathology of patients including early death, failure to thrive and declining motor function. It was also confirmed that fatty liver was recognized earlier. Currently, pathological examination of the central nervous system and microarray analysis are underway. The pathophysiological analysis method using the model fish established by the results of this research may elucidate the pathology of a life-related disease based on abnormal lipid metabolism and develop the therapeutic research, as well as peroxisomal diseases.
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| Free Research Field |
先天代謝異常症
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