2012 Fiscal Year Final Research Report
Development of the next-generation medical treatment for muscularDystrophy
| Project/Area Number |
23659520
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| Research Category |
Grant-in-Aid for Challenging Exploratory Research
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| Allocation Type | Multi-year Fund |
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| Research Field |
Pediatrics
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| Research Institution | Kyoto University |
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Principal Investigator |
HEIKE Toshio 京都大学, 医学研究科, 教授 (90190173)
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| Co-Investigator(Kenkyū-buntansha) |
HIRAMATSU Hidefumi (40362503)
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| Project Period (FY) |
2011 – 2012
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| Keywords | 筋ジストロフィー / iPS 細胞 |
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| Research Abstract |
diseases. Although gene therapy is partly in trial, the standardized therapies are still under investigation. Recently, the preparation procedures for human iPS cells have been set up. By using this strategy, various new studies for new treatments become practicable. For example, the research of small molecules which facilitate exon skipping in disease-responsible region, or which restore the survival of intringically-fragile satellite cells. Recently, we have developed the differentiation procedures intomature muscle cells along with immature muscle cells (or satellite cells as stem cells) in human iPS cells, in addition to in mouse iPS cells (Awaya T, Heike T et al. PloS One 7:e51638 2012）. Simultaneously, we are on the way to make stock of muscular dystrophy-specific iPS cells.
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