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Development of gene therapy for hemolytic anemia using iPS cells

Research Project

Project/Area Number 23790387
Research Category

Grant-in-Aid for Young Scientists (B)

Allocation TypeMulti-year Fund
Research Field Human genetics
Research InstitutionKyushu University

Principal Investigator

MIURA Yoshie  九州大学, 生体防御医学研究所, 学術研究員 (00388935)

Project Period (FY) 2011 – 2012
Project Status Completed (Fiscal Year 2012)
Budget Amount *help
¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000)
Fiscal Year 2012: ¥2,210,000 (Direct Cost: ¥1,700,000、Indirect Cost: ¥510,000)
Fiscal Year 2011: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
Keywords遺伝子治療 / 疾患特異的 iPS 細胞 / 疾患特異的iPS細胞 / 溶血性貧血
Research Abstract

Pyruvate kinase (PK), especially the isozyme PKL and PKR are found in red blood cells, is an important enzyme in red blood cell metabolism. PK deficiency is one of the most common enzymatic defects of red cells. This disorder manifests clinically as anemia. The treatment of this disease includes blood transfusions or the removal of the spleen. However, the treatment usually only reduces the severity of the symptoms and does not cure it. Recently, there have been advances in cell-based therapy that show a lot promise treatment of patients afflicted with genetic and degenerative disorders. However, there are many limitations such as immunological rejection of transplanted tissue, ethical issues such as using embryonicstem cells. Induced pluripotent stem cells (iPS cells) can be established by using 4 transcription factors with mouse somatic cells in vitro. iPS cells can self-renew and differentiate into various types of mature tissue in vitro, so it is considered to be an ideal source for donor tissue. Skin fibroblast was harvested from the wild type miceand PK deficient mice. Then Yamanaka four genes were transduced into skin fibroblast using retrovirus and iPS cells were produced. iPS cells derived from wild type mice and PK deficient mice were differentiated into hematopoietic progenitor-like cells using cytokines and mouse stromal cells. iPS cell technology makes it possible to perform cell transplantation therapies for a wide variety of disease, and it can avoid ethical issues and immune rejection

Report

(3 results)
  • 2012 Annual Research Report   Final Research Report ( PDF )
  • 2011 Research-status Report
  • Research Products

    (18 results)

All 2013 2012 2011

All Journal Article (6 results) (of which Peer Reviewed: 5 results) Presentation (12 results)

  • [Journal Article] Inhibition of PTEN tumor suppressor promotes the generation of induced pluripotent stem cells.2013

    • Author(s)
      Liao J et al
    • Journal Title

      Mol. Ther.

      Volume: (in press)

    • Related Report
      2012 Final Research Report
  • [Journal Article] Inhibition of PTEN tumor suppressor promotes the generation of induced pluripotent stem cells2013

    • Author(s)
      Liao J et al.
    • Journal Title

      Mol Ther.

      Volume: in press

    • Related Report
      2012 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Decreased CXCR3 Expression in CD4+ T Cells Exposed to Asbestos or Derived from Asbestos-exposed Patients2011

    • Author(s)
      Maeda Megumi
    • Journal Title

      American Journal of Respiratory Cell and Molecular Biology

      Volume: 45 Issue: 4 Pages: 795-803

    • DOI

      10.1165/rcmb.2010-0435oc

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Reduction of CXC Chemokine Receptor 3 in an In Vitro Model of Continuous Exposure to Asbestos in a Human T-Cell Line, MT-22011

    • Author(s)
      Maeda Megumi
    • Journal Title

      American Journal of Respiratory Cell and Molecular Biology

      Volume: 45 Issue: 3 Pages: 470-479

    • DOI

      10.1165/rcmb.2010-0213oc

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Decreased CXCR3 expression in CD4+ T cells exposed to asbestos or derived from asbestos-exposed patients.2011

    • Author(s)
      Maeda M, Nishimura Y, Hayashi H, Kumagai N, Chen Y, Murakami S, Miura Y, Hiratsuka J, Kishimoto T, Otsuki T
    • Journal Title

      Am J Resp Cell Mol Biol

      Volume: 45 Pages: 795-803

    • Related Report
      2011 Research-status Report
    • Peer Reviewed
  • [Journal Article] Reduction of CXCR3 in an in vitro model of continuous asbestos exposure on a human T-cell line, MT-2.2011

    • Author(s)
      Maeda M, Nishimura Y, Hayashi H, Kumagai N, Chen Y, Murakami S, Miura Y, Hiratsuka J, Kishimoto T, Otsuki T
    • Journal Title

      Am J Resp Cell Mol Biol

      Volume: 45 Pages: 470-479

    • Related Report
      2011 Research-status Report
    • Peer Reviewed
  • [Presentation] fficient generation of induced pluripotent stem cells by the use of Pten inhibitor. International Society for Stem Cell Research, (ISSCR)2012

    • Author(s)
      Liao J et al
    • Organizer
      0th Annual Meeting
    • Related Report
      2012 Final Research Report
  • [Presentation] SIX IN A SINGLE PARTICLE: NOVEL NON-TRANSMISSIBLE MEASLES VIRUS VECTOR FOR DELIVERY OF SIX GENES ALL TOGETHER.2012

    • Author(s)
      Tahara M et al
    • Organizer
      第18回日本遺伝子治療学会
    • Related Report
      2012 Final Research Report
  • [Presentation] CHARACTERIZATION OF DYSGERMINOMA LIKE TUMORS AROSE IN THE PROCESS OF GENERATING COMMON MARMOSET INDUCED PLURIPOTENT STEM CELLS2012

    • Author(s)
      Yamaguchi S et al.
    • Organizer
      International Society for Stem Cell Research (ISSCR) 10th Annual Meeting
    • Place of Presentation
      横浜
    • Related Report
      2012 Annual Research Report
  • [Presentation] EFFICIENT HEMATOPOIETIC DIFFERENTIATION OF COMMON MARMOSET EMBRYONIC STEM CELLS BY THE INHIBITION OF PI3K-AKT PATHWAY2012

    • Author(s)
      Nii T et al.
    • Organizer
      International Society for Stem Cell Research (ISSCR) 10th Annual Meeting
    • Place of Presentation
      横浜
    • Related Report
      2012 Annual Research Report
  • [Presentation] HIGHLY EFFICIENT GENERATION OF INDUCED PLURIPOTENT STEM CELLS BY THE INHIBITION OF PTEN TUMOR SUPPRESSOR2012

    • Author(s)
      Jiyuan L et al.
    • Organizer
      第18回日本遺伝子治療学会年次学術集会
    • Place of Presentation
      熊本
    • Related Report
      2012 Annual Research Report
  • [Presentation] CHARACTERIZATION OF COMMON MARMOSET DYSGERMINOMA-LIKE TUMORS GENERATED BY THE TRANSDUCTION OF REPROGRAMMING FACTORS2012

    • Author(s)
      Yamaguchi S et al.
    • Organizer
      第18回日本遺伝子治療学会年次学術集会
    • Place of Presentation
      熊本
    • Related Report
      2012 Annual Research Report
  • [Presentation] LYL1, a Basic Helix-Loop-Helix Transcription Factor, Promotes Hematopoietic Differentiation of Common Marmoset Embryonic Stem Cells2011

    • Author(s)
      Kawano H et al
    • Organizer
      American Society of Gene and Cell Therapy 14th Annual Meeting
    • Place of Presentation
      米国
    • Related Report
      2012 Final Research Report
  • [Presentation] Oncogenic risk of induced pluripotent stem cells established from somatic cells with chromosome instability2011

    • Author(s)
      Yamaguchi S et al
    • Organizer
      American Society of Gene and Cell Therapy, 14th Annual MeetingAmerican Society of Gene and Cell Therapy, 14th Annual Meeting
    • Place of Presentation
      米国
    • Related Report
      2012 Final Research Report
  • [Presentation] A basic helix-loop-helix transcription factor Lyl1 regulates hematopoietic differentiation of common marmoset ES cells.2011

    • Author(s)
      Kawano H et al
    • Organizer
      第17回日本遺伝子治療学会
    • Related Report
      2012 Final Research Report
  • [Presentation] Efficient Differentiation of Common Marmoset Embryonic Stem Cells Into Hemangioblast-Like Cells by the Inhibition of PI3K-AKT Pathway2011

    • Author(s)
      Nii T et al
    • Organizer
      53th American Society of Hematology Annual Meeting and Exposition
    • Place of Presentation
      米国
    • Related Report
      2012 Final Research Report
  • [Presentation] LYL1, a Basic Helix-Loop-Helix Transcription Factor, Promotes Hematopoietic Differentiation of Common Marmoset Embryonic Stem Cells.2011

    • Author(s)
      Kawano H, Marumoto T, Okada M, Inoue T, Nii T, Liao J, Yamaguchi S, Nagai Y, Inoue H, Takahashi A, Sasaki E, Miura Y, Tani K
    • Organizer
      American Society of Gene & Cell Therapy 14th Annual Meeting
    • Place of Presentation
      米国シアトル
    • Related Report
      2011 Research-status Report
  • [Presentation] Efficient Differentiation of Common Marmoset Embryonic Stem Cells Into Hemangioblast-Like Cells by the Inhibition of PI3K-AKT Pathway2011

    • Author(s)
      Takenobu Nii, Tomotoshi Marumoto, Hirotaka Kawano, Saori Yamaguchi, Yoko Nagai, Yoshie Miura, Jiyuan Liao, Michiyo Okada, Kenzaburo Tani
    • Organizer
      53rd American Society of Hematology
    • Place of Presentation
      米国サンディエゴ
    • Related Report
      2011 Research-status Report

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Published: 2011-08-05   Modified: 2019-07-29  

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