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Human gene therapy model by induction of stable bone marrow chimerim with regulation of proliferative ability of hematopoietic stem cell

Research Project

Project/Area Number 22590355
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeSingle-year Grants
Section一般
Research Field Experimental pathology
Research InstitutionKitasato University (2011-2012)
The University of Tokyo (2010)

Principal Investigator

TAKEUCHI Yasuo  北里大学, 医学部, 講師 (60286359)

Co-Investigator(Renkei-kenkyūsha) OTSU Makoto  東京大学, 医科学研究所, 准教授 (30361330)
Project Period (FY) 2010 – 2012
Project Status Completed (Fiscal Year 2012)
Budget Amount *help
¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2012: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2011: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2010: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
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 / 造血幹細胞移植 / Lnk 蛋白 / 幹細胞増殖能制御 / 移植前処置軽減 / プラム色素マーカー遺伝子 / 遺伝子導入ウイルス / 遺伝子治療モデル / Lnk遺伝子 / 骨髄造血幹前駆細胞移植 / 細胞内アダプター蛋白
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.

Report

(4 results)
  • 2012 Annual Research Report   Final Research Report ( PDF )
  • 2011 Annual Research Report
  • 2010 Annual Research Report
  • Research Products

    (2 results)

All 2012

All Presentation (2 results)

  • [Presentation] Development of experimental animal model of human gene therapy for X-CGD2012

    • Author(s)
      Takeuchi Y, Amirhosseini M, Franklin L, Takeuchi E, Nakauchi H, Otsu M
    • Organizer
      第38回ヨーロッパ骨髄移植会議
    • Place of Presentation
      Geneva, Switzerland
    • Related Report
      2012 Final Research Report
  • [Presentation] Development of experimental animal model of human gene therapy for X-CGD2012

    • Author(s)
      Yasuo Takeuchi
    • Organizer
      第38回ヨーロッパ骨髄移植会議
    • Place of Presentation
      Geneva, Swiss
    • Related Report
      2012 Annual Research Report

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Published: 2010-08-23   Modified: 2019-07-29  

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