| Project/Area Number |
22890088
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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 |
Human genetics
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| Research Institution | Kyoto University |
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Principal Investigator |
HOTTA Akitsu 京都大学, iPS細胞研究所, 特定拠点助教 (50578002)
|
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| Research Collaborator |
MATSUI Hideto 奈良県立医科大学, 小児科, 講師
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| Project Period (FY) |
2010 – 2011
|
| Project Status |
Completed (Fiscal Year 2011)
|
| Budget Amount *help |
¥3,042,000 (Direct Cost: ¥2,340,000、Indirect Cost: ¥702,000)
Fiscal Year 2011: ¥1,456,000 (Direct Cost: ¥1,120,000、Indirect Cost: ¥336,000)
Fiscal Year 2010: ¥1,586,000 (Direct Cost: ¥1,220,000、Indirect Cost: ¥366,000)
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| Keywords | 遺伝子治療 / iPS細胞 / 遺伝子導入ベクター / 血友病A / 遺伝子導入 / サイレンシング |
|---|
| Research Abstract |
Aiming for developing a novel gene therapy of hemophilia A, we have developed non-viralbased gene delivery vectors that can maintain foreign gene expression for long term. We identified that new transposon vectors were able to maintain reporter gene expression in human iPS cells for up to 150 days. In addition, we report here for the first time that we can deliver the full-length Factor VIII cDNA with 7. 0 kb in size by using our transposon vectors. Our transposon vector is a promising technology for future gene therapy of severe hemophilia A.
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