2012 Fiscal Year Final Research Report
Homologous recombination-mediated gene correction for Adenosine deaminase deficiency
| Project/Area Number |
23791147
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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 |
Pediatrics
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| Research Institution | Tohoku University |
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Principal Investigator |
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| Project Period (FY) |
2011 – 2012
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| Keywords | 原発性免疫不全症 / 遺伝子治療 / 相同組換え / iPS細胞 / 造血幹細胞 |
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| Research Abstract |
iPS cells were derived from skin fibroblasts of ADA deficiency patients. While AAV-mediated gene targeting for the homologous recombination, followed by the selection with deoxyadenosine, was performed, corrected clones were not obtained. iPS cells showed the differentiation into T and B lymphocytes in the culture with OP9 stromal cells. This system is thought to be useful for the functional analysis of corrected ADA gene in iPS cells.
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Author(s)
Moriya K, Suzuki M, Watanabe Y, Takahashi T, Aoki Y, Uchiyama T, Kumaki S, Sasahara Y, Minegishi M, Kure S, Tsuchiya S, Sugamuta K, Ishii N.
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Journal Title
PLos One
Volume: 7(6)
Pages: e37892
DOI
Peer Reviewed
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Eur J Pediatr
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Journal Title
Pediatr. Transplant
Volume: 16(2)
Pages: E63-70
DOI
Peer Reviewed
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