Development of novel gene therapy for congenital blood disorders using induced pluripotent stem cells
| Project/Area Number |
22591071
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Hematology
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| Research Institution | Tokyo Women's Medical University |
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Principal Investigator |
KANNO Hitoshi 東京女子医科大学, 医学部, 准教授 (70221207)
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| Co-Investigator(Kenkyū-buntansha) |
FUJII Hisaichi 東京女子医科大学, 医学部, 教授 (70107762)
UTSUGISAWA Taijyu 東京女子医科大学, 医学部, 助教 (30337133)
谷 憲三朗 九州大学, 生体防御医学研究所, 教授 (00183864)
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| Project Period (FY) |
2010 – 2012
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| Project Status |
Completed (Fiscal Year 2012)
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| Budget Amount *help |
¥3,770,000 (Direct Cost: ¥2,900,000、Indirect Cost: ¥870,000)
Fiscal Year 2012: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2011: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2010: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
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| Keywords | 先天性溶血性貧血 / 動物モデル / 造血幹細胞移植 / 赤血球酵素異常症 / 単一遺伝子疾患 |
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| Research Abstract |
We established an induced pluripotent stem (iPS) cells from murine model of red cell pyruvate kinase (R-PK) deficiency (Pk-1slc). The iPS cells of Pk-1slccould be differentiated into embryoid bodies (EB) and CD71-positive erythroblasts in vitro. Since PKLR gene expressed exclusively in mature erythroid cells, no harmful effect on stem cells as well as progenitor cells has been observed. The iPS cells of Pk-1slc are useful for both hematopoietic stem cell transplantation and screening of novel therapeutic drugs for causative therapy of human PK deficiency associated with congenital hemolytic anemia
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Report
(4 results)
Research Products
(56 results)
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[Journal Article] Extensive gene deletions in Japanese patients with Diamond-Blackfan anemia2011
Author(s)
Kuramitsu M, Matsubara A, Morio T, Takagi M, Toki T, Terui K, RuNan W, Kanno H, Ohga S, Ohara A, Kitoh T, Sugita K, Kudo K, Matsubayashi T, Mizue N, Ozeki M, Masumi A, Momose H, Takizawa K, Mizukami T, Yamaguchi K, Ogawa S, Ito E, Hamaguchi I
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Journal Title
Blood
Volume: (in press)
Issue: 10
Pages: 2376-84
DOI
Related Report
Peer Reviewed
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[Presentation] 先天性溶血性貧血2011
Author(s)
菅野仁
Organizer
第56回日本未熟児新生児学会学術集会
Place of Presentation
東京国際フォーラム(招待講演)
Year and Date
2011-11-15
Related Report
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