| Project/Area Number |
26670269
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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 |
Applied pharmacology
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| Research Institution | The University of Tokushima |
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Principal Investigator |
ITOH Kohji 徳島大学, 大学院医歯薬学研究部, 教授 (00184656)
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| Co-Investigator(Kenkyū-buntansha) |
TSUJI Daisuke 徳島大学, 大学院医歯薬学研究部, 助教 (00423400)
IKUO Mariko 徳島大学, 大学院医歯薬学研究部, 特任助教 (60713401)
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| Co-Investigator(Renkei-kenkyūsha) |
URANO Yasuteru 東京大学, 医学(系)研究科(研究院), 教授 (20292956)
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| Project Period (FY) |
2014-04-01 – 2016-03-31
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| Project Status |
Completed (Fiscal Year 2015)
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| Budget Amount *help |
¥3,770,000 (Direct Cost: ¥2,900,000、Indirect Cost: ¥870,000)
Fiscal Year 2015: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Fiscal Year 2014: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
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| Keywords | ダイレクトリプログラミング / 疾患iPS細胞 / induced Neuron (iN細胞） / リソソーム病 / 蛍光イメージング / トラフィッキング / トラフィキング / ダイいレクトリプログラミング |
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| Outline of Final Research Achievements |
This study was performed to establish induced Neurons (iN cells) by direct reprogramming from patient fibroblasts and/or induce neurons from the iPS cells derived from the patient with lysosomal diseases involving neurological symptoms. We succeeded in inducing cortical neurons (CN) from the patient iPS cells with Tay-Sachs disease (TSD, Hexαdeficiency) and cathepsin A (CTSA) deficiency, in which their substrate accumulation was observed. The TSD-CN showed lower cell viability in the absence of neurotrophic factors, that was restored by normal recombinant Hex enzyme replacement. The CN culture system induced from the iPS cells with neurodegenerative lysosomal diseases was considered to be useful for studying the neuropathogenesis and evaluating therapeutic approaches.
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