Analysis of abnormal splicing mechanism and application for thetreatment of Muscular Dystrophy
Project/Area Number |
22603002
|
Research Category |
Grant-in-Aid for Scientific Research (C)
|
Allocation Type | Single-year Grants |
Section | 一般 |
Research Field |
ケミカルバイオロジー
|
Research Institution | Kyoto University (2011-2012) Tokyo Medical and Dental University (2010) |
Principal Investigator |
|
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)
|
Project Period (FY) |
2010 – 2012
|
Project Status |
Completed (Fiscal Year 2012)
|
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.
|
Report
(4 results)
Research Products
(28 results)
-
-
-
[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
-
Journal Title
Sci. Rep
Volume: 1
Issue: 1
Pages: 92-92
DOI
Related Report
Peer Reviewed
-
[Journal Article] Chemical treatment enhances skipping of a mutated 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
-
Journal Title
Nat Commun
Volume: 2
Issue: 1
Pages: 308-308
DOI
NAID
Related Report
Peer Reviewed
-
[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
Related Report
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-