Establishment of novel cellular therapy for hemophilia A using autologous blood outgrowth endothelial progenitors
Project/Area Number |
22790995
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Research Category |
Grant-in-Aid for Young Scientists (B)
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Allocation Type | Single-year Grants |
Research Field |
Pediatrics
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Research Institution | Nara Medical University |
Principal Investigator |
MATSUI Hideto 奈良県立医科大学, 医学部, 講師 (00571027)
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Project Period (FY) |
2010 – 2011
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Project Status |
Completed (Fiscal Year 2011)
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Budget Amount *help |
¥3,900,000 (Direct Cost: ¥3,000,000、Indirect Cost: ¥900,000)
Fiscal Year 2011: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Fiscal Year 2010: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
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Keywords | 小児血液学 / 血友病A / 血管内皮前駆細胞 / レンチウイルスベクター / 遺伝子治療 / 細胞療法 / イヌモデル |
Research Abstract |
We have established ex vivo gene transfer system using genetically-modified blood outgrowth endothelial progenitor cells(BOECs) which are capable of synthesizing therapeutic levels of plasma FVIII over a prolonged time periods.^<20> Using this ex vivo gene transfer strategy, we can provide for the safe and efficacious delivery of FVIII following subcutaneous implantation of BOECs in hemophilia A mouse model. Now, we would like to try this strategy in hemophilia A dog model.
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Report
(3 results)
Research Products
(15 results)
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[Journal Article] A microRNA-regulated and GP64-pseudotyped lentiviral vectors mediated stable expression of FVIII in murine model of hemophilia A2011
Author(s)
Matsui H, Hegadorn C, Ozelo M, Burnett E, Tuttle A, Labelle A, McCray PB Jr, Naldini L, Brown B, Hough C, Lillicrap D
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Journal Title
Molecular Therapy
Volume: 19(4)
Pages: 723-730
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