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Preparation and application of mouse model for the study on chaperone therapy for Fabry disease

Research Project

Project/Area Number 20590300
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeSingle-year Grants
Section一般
Research Field Pathological medical chemistry
Research InstitutionObihiro University of Agriculture and Veterinary Medicine

Principal Investigator

ISHII Satoshi  Obihiro University of Agriculture and Veterinary Medicine, 医学部, 客員研究員 (00222935)

Co-Investigator(Kenkyū-buntansha) MATSUDA Junichiro  (独)医薬基盤研究所, 生物資源研究部, 研究リーダー (60181731)
Project Period (FY) 2008 – 2010
Project Status Completed (Fiscal Year 2010)
Budget Amount *help
¥4,810,000 (Direct Cost: ¥3,700,000、Indirect Cost: ¥1,110,000)
Fiscal Year 2010: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000)
Fiscal Year 2009: ¥910,000 (Direct Cost: ¥700,000、Indirect Cost: ¥210,000)
Fiscal Year 2008: ¥2,860,000 (Direct Cost: ¥2,200,000、Indirect Cost: ¥660,000)
Keywordsファブリー病 / シャペロン療法 / 応用動物
Research Abstract

The purpose of our study is to establish a mouse model for the chaperone therapy for Fabry disease. Fabry disease is an inherited disease caused by the deficient activity of α-galactosidase A and resulted to accumulation of glycosphingolipid, predominantly globotriaosylceramide (Gb3). The mouse model established previously was good for the determination of enzyme activity but not for the accumulation of Gb3 on the effect of drug candidates. In our present study, we prepared the transgenic mouse expressing human Gb3 synthase gene, and we succeeded a mouse model in which we can determine the effect of compounds on both enzyme activity and Gb3 content.

Report

(4 results)
  • 2010 Annual Research Report   Final Research Report ( PDF )
  • 2009 Annual Research Report
  • 2008 Annual Research Report
  • Research Products

    (9 results)

All 2011 2010 2009 Other

All Journal Article (4 results) (of which Peer Reviewed: 4 results) Presentation (2 results) Book (2 results) Remarks (1 results)

  • [Journal Article] Increased globotriaosylceramide levels in a transgenic mouse expressing human α 1,4-galactosyl-transferase and mouse model for Fabry disease2011

    • Author(s)
      Shiozuka C., Mastuda J., Ishii S.
    • Journal Title

      Journal of Biochemistry 149

      Pages: 161-170

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Increased globotriaosylceramide levels in a transgenic mouse expressing human α1,4-galactosyltransferase and a mouse model for Fabry disease2011

    • Author(s)
      Shiozuka C., et al.
    • Journal Title

      Journal of Biochemistry

      Volume: 149 Pages: 161-170

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Preclinical efficacy and safety of 1-deoxygalacto-nojirimycin in mice for Fabry disease2009

    • Author(s)
      Ishii S., et al.
    • Journal Title

      The journal of pharmacology and experimental therapeutics 328

      Pages: 723-731

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Preclinical efficacy and safety of 1-deoxygalactonojirimycin in mice for Fabry disease2009

    • Author(s)
      Ishii S, et.al.
    • Journal Title

      The Journal of pharmacology and experimental therapeutics 328

      Pages: 723-731

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Presentation] Gb3の高度蓄積により発症するファブリー病病態モデルマウス2010

    • Author(s)
      田口惇美
    • Organizer
      第33回日本分子生物学会年会・第83回日本生化学会大会合同大会
    • Place of Presentation
      神戸ポートアイランド(兵庫県)
    • Year and Date
      2010-12-10
    • Related Report
      2010 Annual Research Report 2010 Final Research Report
  • [Presentation] 1-デオキシガラクトノジリマイシンはファブリー病モデルマウス組織において糖脂質の蓄積を低下させる2009

    • Author(s)
      田口惇美
    • Organizer
      第82回日本生化学会大会
    • Place of Presentation
      神戸ポートアイランド(兵庫県)
    • Year and Date
      2009-10-24
    • Related Report
      2010 Final Research Report 2009 Annual Research Report
  • [Book] Pharmacological chaperone therapy for Fabry disease2010

    • Author(s)
      Fan J.Q., Ishii S.
    • Publisher
      Springer Science+Business Media、Fabry Disease Chapter
    • Related Report
      2010 Final Research Report
  • [Book] Fabry Disease, Chapter 29 : Pharmacological chaperone therapy for Fabry disease2010

    • Author(s)
      Fan J.Q., Ishii S.
    • Publisher
      Springer Science+Business Media
    • Related Report
      2010 Annual Research Report
  • [Remarks] ホームページ等

    • Related Report
      2010 Final Research Report

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Published: 2008-04-01   Modified: 2016-04-21  

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