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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| 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: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2016: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2015: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
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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 / 診断システム / 全エクソーム解析 / ペルオキシソーム / iPS細胞 / ゼブラフィッシュ / 神経発生異常 |
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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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Report
(4 results)
Research Products
(21 results)
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[Journal Article] Profiling and Imaging of Phospholipids in Brains of Abcd1-Deficient Mice2018
Author(s)
Hama K, Fujiwara Y, Morita M, Yamazaki F, Nakashima Y, Takei S, Takashima S, Setou M, Shimozawa N, Imanaka T, Yokoyama K.
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Journal Title
Lipids
Volume: 53
Pages: 85-102
DOI
Related Report
Peer Reviewed / Open Access
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[Journal Article] Ataxic form of autosomal recessive PEX10-related peroxisome biogenesis disorders with a novel compound heterozygous gene mutation and characteristic clinical phenotype2017
Author(s)
Toru Yamashita, Jun Mitsui, Nobuyuki Shimozawa, Shigeo Takashima, Hiroshi Umemurad, Kota Sato, Mami Takemoto, Nozomi Hishikawa, Yasuyuki Ohta, Takashi Matsukawa, Hiroyuki Ishiura, Jun Yoshimura, Koichiro Doi, Shinichi Morishita, Shoji Tsuji, Koji Abe.
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Journal Title
J Neurol Sci
Volume: 375
Pages: 424-429
DOI
Related Report
Peer Reviewed
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