2012 Fiscal Year Final Research Report
Development of gene therapy for hemolytic anemia using iPS cells
| Project/Area Number |
23790387
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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 |
Human genetics
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| Research Institution | Kyushu University |
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Principal Investigator |
MIURA Yoshie 九州大学, 生体防御医学研究所, 学術研究員 (00388935)
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| Project Period (FY) |
2011 – 2012
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| Keywords | 遺伝子治療 / 疾患特異的 iPS 細胞 |
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| Research Abstract |
Pyruvate kinase (PK), especially the isozyme PKL and PKR are found in red blood cells, is an important enzyme in red blood cell metabolism. PK deficiency is one of the most common enzymatic defects of red cells. This disorder manifests clinically as anemia. The treatment of this disease includes blood transfusions or the removal of the spleen. However, the treatment usually only reduces the severity of the symptoms and does not cure it. Recently, there have been advances in cell-based therapy that show a lot promise treatment of patients afflicted with genetic and degenerative disorders. However, there are many limitations such as immunological rejection of transplanted tissue, ethical issues such as using embryonicstem cells. Induced pluripotent stem cells (iPS cells) can be established by using 4 transcription factors with mouse somatic cells in vitro. iPS cells can self-renew and differentiate into various types of mature tissue in vitro, so it is considered to be an ideal source for donor tissue. Skin fibroblast was harvested from the wild type miceand PK deficient mice. Then Yamanaka four genes were transduced into skin fibroblast using retrovirus and iPS cells were produced. iPS cells derived from wild type mice and PK deficient mice were differentiated into hematopoietic progenitor-like cells using cytokines and mouse stromal cells. iPS cell technology makes it possible to perform cell transplantation therapies for a wide variety of disease, and it can avoid ethical issues and immune rejection
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Research Products
(9 results)
