Development of a novel treatment for SMA using SMA-iPSCs disease models with different genetic background
Project/Area Number |
16K10008
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Research Category |
Grant-in-Aid for Scientific Research (C)
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Allocation Type | Multi-year Fund |
Section | 一般 |
Research Field |
Pediatrics
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Research Institution | 独立行政法人国立病院機構長良医療センター(臨床研究部) |
Principal Investigator |
FUNATO Michinori 独立行政法人国立病院機構長良医療センター(臨床研究部), 長良医療センター臨床研究部, 第二小児科医長 (30420350)
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Research Collaborator |
OHUCHI Kazuki
ANDO Shiori
SATO Arisu
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Project Period (FY) |
2016-04-01 – 2019-03-31
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Project Status |
Completed (Fiscal Year 2018)
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Budget Amount *help |
¥4,680,000 (Direct Cost: ¥3,600,000、Indirect Cost: ¥1,080,000)
Fiscal Year 2018: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2017: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2016: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
|
Keywords | 疾患モデル / iPS細胞 / 薬剤応答性 / 治療薬開発 / 創薬 / 薬剤反応性 / 神経科学 / 再生医学 |
Outline of Final Research Achievements |
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder characterized by the degeneration of spinal motor neurons. This disease is caused by the deletion of the survival motor neuron protein. Currently, there are no curative agents for SMA, although some research groups have developed treatments based on the molecular pathophysiology of this disease. Our purpose in this study is to establish a number of human SMA with different genetic background-derived induced pluripotent stem cells (SMA-iPSCs) disease model and develop a standard treatment for SMA. Before now, we established SMA-iPSCs disease models derived from 19 SMA patients with different genetic background. In addition, we assayed a therapeutic drug of thyrotropin-releasing hormone (TRH) analog, a free radical scavenger (edaravone), a docosahexaenoic acid, γ-secretase inhibitor, and rufinamide.
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Academic Significance and Societal Importance of the Research Achievements |
SMAは難治性の希少疾患ではあるが、背景にひそむ病態は多種多様である。本研究によって、薬剤の応答性の個人差が解明されれば、希少疾患におけるテーラーメイド医療へのさらなる道筋を提示することになる。
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Report
(4 results)
Research Products
(10 results)
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[Journal Article] The Protective Effects of Levetiracetam on a Human iPSCs-Derived Spinal Muscular Atrophy Model.2019
Author(s)
Ando S, Funato M, Ohuchi K, Inagaki S, Sato A, Seki J, Kawase C, Saito T, Nishio H, Nakamura S, Shimazawa M, Kaneko H, Hara H.
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Journal Title
Neurochem Res.
Volume: in press
Issue: 7
Pages: 1773-1779
DOI
Related Report
Peer Reviewed / Open Access
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[Journal Article] A docosahexaenoic acid-derived pro-resolving agent, maresin 1, protects motor neuron cells death2018
Author(s)
Ohuchi K., Ono Y., Joho M., Tsuruma K., Ogami S., Yamane S., Funato M., Kaneko H., Nakamura S., Hara H. and Shimazawa M
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Journal Title
Neurochemical Research
Volume: 43(7)
Issue: 7
Pages: 1413-1423
DOI
Related Report
Peer Reviewed / Open Access
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[Journal Article] Edaravone is a candidate agent for spinal muscular atrophy: In vitro analysis using a human induced pluripotent stem cells-derived disease model.2017
Author(s)
Ando S, Funato M, Ohuchi K, Kameyama T, Inagaki S, Seki J, Kawase C, Tsuruma K, Shimazawa M, Kaneko H, Hara H.
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Journal Title
Eur J Pharmacol.
Volume: 814
Pages: 161-168
DOI
Related Report
Peer Reviewed / Open Access
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