Disease modeling of genetic muscular disorders using patient-derived induced pluripotent stem cells
| Project/Area Number |
25870375
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| Research Category |
Grant-in-Aid for Young Scientists (B)
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| Allocation Type | Multi-year Fund |
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| Research Field |
Pediatrics
Neurochemistry/Neuropharmacology
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| Research Institution | Kyoto University |
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Principal Investigator |
AWAYA Tomonari 京都大学, 医学(系)研究科(研究院), 助教 (20589593)
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| Project Period (FY) |
2013-04-01 – 2015-03-31
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| Project Status |
Completed (Fiscal Year 2014)
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| Budget Amount *help |
¥4,290,000 (Direct Cost: ¥3,300,000、Indirect Cost: ¥990,000)
Fiscal Year 2014: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2013: ¥2,600,000 (Direct Cost: ¥2,000,000、Indirect Cost: ¥600,000)
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| Keywords | iPS細胞 / デュシェンヌ型筋ジストロフィー / ポンペ病 / 病態再現 / 神経分化 / 心筋分化 / 骨格筋分化 / 中枢神経分化 / 疾患iPS細胞 / 遺伝性筋疾患 / 筋ジストロフィー |
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| Outline of Final Research Achievements |
We aimed to provide in vitro disease models of genetic muscular disorders using patient-derived induced pluripotent stem (iPS) cells. We established patient-derived iPS cell lines from 9 Duchenne muscular dystrophy (DMD) patients and 3 Pompe disease patients. We conducted neuronal, cardiac muscular, and skeletal muscular differentiation of the cells to compare in vitro characteristics to those from wild-type iPS cells. Based on the results, we are now developing drug-screening platforms.
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Report
(3 results)
Research Products
(5 results)
