2014 Fiscal Year Final Research Report
Expression of dystrophin via exon skipping with a small chemical
Project/Area Number |
24390267
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Research Category |
Grant-in-Aid for Scientific Research (B)
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Allocation Type | Partial Multi-year Fund |
Section | 一般 |
Research Field |
Pediatrics
|
Research Institution | Kobe Gakuin University |
Principal Investigator |
MATSUO Masafumi 神戸学院大学, 総合リハビリテーション学部, 教授 (10157266)
|
Project Period (FY) |
2012-04-01 – 2015-03-31
|
Keywords | Duchenne型筋ジストロフィー / エクソンスキッピング / 低分子 |
Outline of Final Research Achievements |
Antisense-meditated exon skipping is the most promising treatment for Duchenne muscular dystrophy. Here, small chemicals that induce dystrophin exon skipping were searched using in vitro splicing system. Unfortunately, chemicals that can be applied for clinical use were not identified yet. Furthermore, a new splicing analysis system that enabled detection of skipping of 10 exons simultaneously was established to enhance screening of chemicals.
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Free Research Field |
小児科
|