2014 Fiscal Year Final Research Report
Study of the gene therapy for pyruvate kinase abnormality as a model of therapy for serious heredity erythrocyte disease.
| Project/Area Number |
25893164
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| Research Category |
Grant-in-Aid for Research Activity Start-up
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| Allocation Type | Single-year Grants |
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| Research Field |
Pediatrics
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| Research Institution | Kyushu University |
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Principal Investigator |
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| Project Period (FY) |
2013-08-30 – 2015-03-31
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| Keywords | 遺伝子治療 / 赤血球異常 / 造血幹細胞 / iPS細胞 |
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| Outline of Final Research Achievements |
We examined the utility of the pyruvate kinase deficiency (PKD) mouse origin iPS (PKD-iPS) cell towards development of the new gene therapy to PKD.
In the established PKD-iPS cell, we found the same DNA variation that is existed in the cells of model mouse. The iPS cells were be able to differentiate to the erythroid cells, but the number of erythrocytes were decreased compared with cells from the wild-type iPS cells. Furthermore, we made the guide RNA / Cas9 gene expression vector and the donor vector for repairing the varied PKLR gene. To transfer these vectors into iPS cells efficiently, we examined the transgene efficiency, and get transfer efficiency 50% that is exceeding 40% of the transgene efficiency needed for genome edit.
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| Free Research Field |
遺伝子治療
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