Analysis of abnormal splicing mechanism and application for thetreatment of Muscular Dystrophy
Project/Area Number |
22603002
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Research Category |
Grant-in-Aid for Scientific Research (C)
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Allocation Type | Single-year Grants |
Section | 一般 |
Research Field |
ケミカルバイオロジー
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Research Institution | Kyoto University (2011-2012) Tokyo Medical and Dental University (2010) |
Principal Investigator |
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Co-Investigator(Renkei-kenkyūsha) |
HAGIWARA Masatoshi 京都大学, 医学研究科形態形成機構学, 講座 (10208423)
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Project Period (FY) |
2010 – 2012
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Project Status |
Completed (Fiscal Year 2012)
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Budget Amount *help |
¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2012: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2011: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000)
Fiscal Year 2010: ¥2,210,000 (Direct Cost: ¥1,700,000、Indirect Cost: ¥510,000)
|
Keywords | スプライシング / ジストロフィン / 筋ジストロフィー / 低分子化合物 / レポーター |
Research Abstract |
We have tried to set up the treatment of Duchenne Muscular Dystrophy by promoting mutated exon skipping by using a chemical compound. We found that TG003, a specific inhibitor for Clk kinases, promotes exon skipping in the patient cells. We also comprehensively analyzed the effect of TG003 in pre-mRNA splicing and detected the introns whose excision is affected.
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Report
(4 results)
Research Products
(29 results)
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[Journal Article] Specific Y14 domains mediate its nucleo-cytoplasmic shuttling and association with spliced mRNA2011
Author(s)
Kataoka, N., , Diem, M.D., Yoshida, M., Hatai, C., Dobashi, I., Dreyfuss, G, Hagiwara, M. and Ohno, M
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Journal Title
Scientific Reports
Volume: 1
Pages: 92
DOI
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[Journal Article] Chemical treatment enhances skipping of amutated exon in the dystrophin gene2011
Author(s)
Nishida, A., Kataoka,N., Takeshima. Y., Yagi, M. Awano, H., Ota, M., Itoh, K., Hagiwara, M. and Matsuo, M
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Journal Title
Nature Communications
Volume: 2
Pages: 308
DOI
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[Presentation] Modulators for splicing of IKBKAP gene, a responsible gene for Familial dysautonomia2012
Author(s)
Kataoka, N., Yoshida, M., Usui, T., Sakuma, M. and Hagiwara, M
Organizer
Cold Spring Harbor Asia 2012 Conference on RNA Biology
Place of Presentation
Suzhou, China
Year and Date
20120000
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