| Project/Area Number |
23591496
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | Kobe University |
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Principal Investigator |
YAGI Mariko 神戸大学, 医学(系)研究科(研究院), 研究員 (60362787)
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| Co-Investigator(Kenkyū-buntansha) |
TAKESHIMA Yasuhiro 神戸大学, 大学院医学研究科, 教授 (40281141)
AWANO Hiroyuki 神戸大学, 大学院医学研究科, 特命助教 (30437470)
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| Project Period (FY) |
2011 – 2013
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| Project Status |
Completed (Fiscal Year 2013)
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| Budget Amount *help |
¥5,070,000 (Direct Cost: ¥3,900,000、Indirect Cost: ¥1,170,000)
Fiscal Year 2013: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2012: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2011: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
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| Keywords | スプライシング / 国際情報研究 / 国際情報交流 |
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| Research Abstract |
Antisense oligonucleotides that induce exon skipping is now attracting much attention to express internally deleted-dystrophin in Duchenne muscular dystrophy(DMD). Previously, we identified antisense oligonucleotides(AO85) that could induce exon 45 skipping efficiently. We examined the ability of AO85 to induce exon 45 skipping and dystrophin expression in DMD patient-derived myocytes carrying different types of deletion mutations, as follows:exon 46-47, 46-48, 46-49, 46-51 or 46-53. The skipping efficiency was different from patients to patients. We identified the junction site of each patient with deletion mutation and examined the intronic splicing enhancers/silencers (ISEs/ISSs) in the junction site. In this study, we could not clarify the correlation between ISEs/ISSs in the junction site and exon 45 skipping efficiency.
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