Functional analysis to reveal the role of GATA2 during hematopoietic differentiation based on disease-specific iPS cells

2017 Fiscal Year Final Research Report

• Project/Area Number: 16K19564
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Hematology
• Research Institution: Tohoku University
• Principal Investigator: HATTA SHUNSUKE 東北大学, 医学系研究科, 非常勤講師 (80772194)
• Project Period (FY): 2016-04-01 – 2018-03-31
• Keywords: 血液内科学

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.

Free Research Field

医歯薬学