| Budget Amount *help |
¥3,900,000 (Direct Cost: ¥3,000,000、Indirect Cost: ¥900,000)
Fiscal Year 2017: ¥2,470,000 (Direct Cost: ¥1,900,000、Indirect Cost: ¥570,000)
Fiscal Year 2016: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
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| Outline of Final Research Achievements |
We aimed to establish induced pluripotent stem (iPS) cells using episomal vectors based on patient-derived mesenchymal stem cells. So far, we have not succeeded to establish the iPS cells, presumably due to increased genome instability caused by GATA-2 mutation. Alternatively, we established iPS cells from patient with X-linked sidroblastic anemia (XLSA). When the iPS cells were differentiated into erythroid cells by co-culturing with stromal cells, the erythroblasts exhibited ringed sideroblasts and abnormal iron deposition in the mitochondria, confirmed by electron microscopy. Further phenotypic analyses would lead to the better understanding of the molecular basis of XLSA as well as the establishment of novel therapeutic strategies.
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