Functional Analysis of iPSC-derived Myocytes from a Patient with Carnitine Palmitoyltransferase II Deficiency
| Project/Area Number |
25870995
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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
Metabolomics
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| Research Institution | Fukuoka University |
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Principal Investigator |
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| Project Period (FY) |
2013-04-01 – 2016-03-31
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| Project Status |
Completed (Fiscal Year 2015)
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| Budget Amount *help |
¥4,290,000 (Direct Cost: ¥3,300,000、Indirect Cost: ¥990,000)
Fiscal Year 2014: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Fiscal Year 2013: ¥2,470,000 (Direct Cost: ¥1,900,000、Indirect Cost: ¥570,000)
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| Keywords | iPS / CPT II deficiency / iPS細胞 / 先天性横紋筋融解症 / 横紋筋融解症 / CPT２欠損症 / CPTII欠損症 |
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| Outline of Final Research Achievements |
We induced differentiation of CPT II-deficient hiPSCs into mature myocytes in a highly efficient and reproducible manner and recapitulated some aspects of the disease phenotypes of CPT II deficiency in the myocyte disease models. This approach addresses the challenges of modeling the abnormality of FAO in CPT II deficiency using iPSC technology and has the potential to revolutionize translational research in this field.
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Report
(4 results)
Research Products
(2 results)
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[Journal Article] Functional Analysis of iPSC-derived Myocytes from a Patient with Carnitine Palmitoyltransferase II Deficiency2014
Author(s)
Tetsuhiko Yasuno, Kenji Osafune, Hidetoshi Sakurai, Isao Asaka, Akihito Tanaka, Seiji Yamaguchi, Kenji Yamada, Hirofumi Hitomi, Sayaka Arai, Yuko Kurose, Yasuki Higaki, Mizuki Sudo, Souichi Ando, Hitoshi Nakashima, Takao Saito, Hidetoshi Kaneoka
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Journal Title
Biochem. and Biophys. Res. Commun
Volume: (in press)
Issue: 2
Pages: 175-181
DOI
Related Report
Peer Reviewed / Open Access
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