Establishment of induced neurons (iN cells) derived from lysosomal disease patients involving neurological symptoms and elucidation of regulatory mechanism of neurodegeneration
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 |
Research Field |
Applied pharmacology
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Research Institution | The University of Tokushima |
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)
|
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 |
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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Report
(3 results)
Research Products
(53 results)
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[Journal Article] Protease-resistant modified human β-hexosaminidase B ameliorates symptoms in GM2 gangliosidosis model.2016
Author(s)
Kitakaze K, Mizutani Y, Sugiyama E, Tasaki C, Tsuji D, Maita N, Hirokawa T, Asanuma D, Kamiya M, Sato K, Setou M, Urano Y, Togawa T, Otaka A, Sakuraba H, Itoh K
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Journal Title
J Clin Invest
Volume: in press
Issue: 5
Pages: 1691-1703
DOI
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Peer Reviewed / Open Access / Int'l Joint Research
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[Journal Article] Pim-2 kinase is an important target of treatment for tumor progression and bone loss in myeloma.2015
Author(s)
Hiasa M, Teramachi J, Oda A, Amachi R, Harada T, Nakamura S, Miki H, Fujii S, Kagawa K, Watanabe K, Endo I, Kuroda Y, Yoneda T, Tsuji D et al.
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Journal Title
Leukemia
Volume: 29
Issue: 1
Pages: 207-217
DOI
Related Report
Peer Reviewed / Open Access
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[Journal Article] In vivo crystallography at X-ray free-electron lasers: the next generation of structural biology?2014
Author(s)
Gallat Francois-Xavier, N. Matsugaki, P. Coussens Nathan, Yagi J. K., Boudes M., T. Higashi, Daisuke Tsuji, Tatano Yutaka, M. Suzuki, E. Mizohata, K. Tono, Y. Hatui, M. Yabashi, E. Nango, K.Itou, Coulibaly Fasseli, Tobe Stephen, Ramaswamy S., S. Iwata and Chavas M. L.
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Journal Title
The Royal Society
Volume: 369
Issue: 1647
Pages: 20130497-20130497
DOI
Related Report
Peer Reviewed / Open Access / Acknowledgement Compliant
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[Presentation] グリア細胞における ABCA7 発現制御機構の検討2016
Author(s)
市野 晨人, 奥平 桂一郎, 川原 遥華, 木村 仁, 辻 大輔, 西辻 和親, 堂前 純子, 道川 誠 坂下 直実, 伊藤 孝司, 斎藤 博幸.
Organizer
日本薬学会第136年会
Place of Presentation
神奈川県横浜市(パシフィコ横浜)
Year and Date
2016-03-28
Related Report
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[Presentation] リソソーム病におけるオートリソソームの形成異常2015
Author(s)
辻 大輔, 本窪田 絢加, 北風 圭介, 山口 沙恵香, 田崎 智佳子, 伊藤 孝司.
Organizer
BMB2015 第38回日本分子生物学会年会・第88回日本生化学会大会 合同大会
Place of Presentation
兵庫県神戸市(神戸国際会議場)
Year and Date
2015-12-03
Related Report
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[Presentation] タンパク質半化学合成を指向した配列特異的チオエステル化法の開発2015
Author(s)
津田 雄介, 重永 章, 辻 耕平, 傳田 将也, 佐藤 浩平, 北風 圭介, 中村 太寛, 猪熊 翼, 伊藤 孝司, 大髙 章
Organizer
創薬懇話会2015 in 徳島 次世代を担う若手のためのメディシナルケミストリーフォーラム
Place of Presentation
徳島県鳴門市(グランドエクシブ鳴門 ザ・ロッジ)
Year and Date
2015-07-02
Related Report
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[Presentation] 新規組換えリソソーム酵素の創製とリソソーム病治療への応用2015
Author(s)
伊藤 孝司, 西岡 宗一郎, 辻 大輔, 東 哲也, 真板 宣男, 小林 功, 瀬筒 秀樹, 湯本 史明, 原園 景, 石井 明子, 川崎 ナナ
Organizer
第15回蛋白質科学会年会
Place of Presentation
徳島県徳島市(あわぎんホール)
Year and Date
2015-06-26
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[Presentation] Development of Chemistry-based Protocol for Sequence-dependent Thioesterification.2015
Author(s)
Yusuke Tsuda, Akira Shigenaga, Kohei Tsuji, Masaya Denda, Kohei Sato, Keisuke Kitakaze, Takahiro Nakamura, Tsubasa Inokuma, Kohji Itoh, Akira Otaka
Organizer
American Peptide Symposium 2015
Place of Presentation
Hyatt Regency (Orland, Florida, USA)
Year and Date
2015-06-20
Related Report
Int'l Joint Research
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[Presentation] Development of protease-resistant modified human β-hexosaminidase B and evaluation of intracerebroventricular replacement effects on GM2 gangliosidosis model mice.2015
Author(s)
Kitakaze, K., Tasaki, C., Mizutani,Y. Sugiyama, E., Ikuo, M., Kamiya, M., Setou, M., Urano, Y. and Itoh, K.
Organizer
The 11th Annual World Symposium
Place of Presentation
Hyatt Regency Orlando(USA,Orlando)
Year and Date
2015-02-10
Related Report
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[Book] 癌と化学療法 - 術前2週間のS-1投与によりGrade2の治療効果が得られた進行胃癌の1例-2015
Author(s)
湯浅 康弘, 沖津 宏, 後藤 正和, 枝川 広志, 大森 理佐, 谷 亮太郎, 増田 有理, 蔵本 俊輔,松本 大資, 富林 敦司, 浜田 陽子, 山下 留理子, 藤井 義幸, 伊藤 孝司
Total Pages
120
Publisher
株式会社 癌と化学療法社
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