2012 Fiscal Year Final Research Report
Human gene therapy model by induction of stable bone marrow chimerim with regulation of proliferative ability of hematopoietic stem cell
| Project/Area Number |
22590355
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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 |
Experimental pathology
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| Research Institution | Kitasato University (2011-2012)
The University of Tokyo (2010) |
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Principal Investigator |
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| Co-Investigator(Renkei-kenkyūsha) |
OTSU Makoto 東京大学, 医科学研究所, 准教授 (30361330)
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| Project Period (FY) |
2010 – 2012
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| Keywords | 造血幹細胞 / 遺伝子治療 / Lnk 遺伝子変異体 / 骨髄キメリズム / 遺伝子導入 / 慢性肉芽腫症 |
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| Research Abstract |
The gene therapy using hematopoietic stem cells (HSCs) is a curative for X-linked chronic granulomatous disease. However, long-term treatment result is not satisfactory because of disappearance of neutrophils with functional reconstitution. We attempt the development of curative gene therapy model by transductionof Lnk-gene variant which promotes appropriate proliferation into HSCs. We made an animal gene therapy model with reproducing clinical problem of human gene therapy. But, the confirmation of the efficacy of transduction or expression of Lnk variant is difficult. We reexamine the modification of a gene vector or methods for transduction.
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