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2012 Fiscal Year Final Research Report

Gene Therapy for Fabry disease: development of a miniature swine model

Research Project

  • PDF
Project/Area Number 22590303
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeSingle-year Grants
Section一般
Research Field Human genetics
Research InstitutionKagoshima University

Principal Investigator

TAKENAKA Toshihiro  鹿児島大学, 大学院・医歯学総合研究科, 特任准教授 (70363645)

Co-Investigator(Kenkyū-buntansha) HIGUCHI Koji  鹿児島大学, 大学院・医歯学総合研究科, 特任助教 (90448580)
YOSHIMITSU Makoto  鹿児島大学, 大学院・医歯学総合研究科, 特任助教 (70404530)
Project Period (FY) 2010 – 2012
Keywords遺伝子治療 / 心 Fabry 病 / Fabry 病 / ミニブタ / 造血幹細胞
Research Abstract

The aim of this study is to develop a miniature swine model forgene therapy for Fabry disease. We tried to collect hematopoietic stem cells from bonemarrow or G-CSF mobilized peripheral blood mononuclear cells, however G-CSF mobilized apherisis method only allowed us to collect enough number of peripheral blood mononuclear cells. We also tried to establish mouse anti-miniature swine CD34 peptide antibody, however the cells recognized by the antibody did not grow in cytokine containing methlycellulose plate. Further characterization of miniature swine hematopoietic stem cell need to be clarified for utilizing this model in gene therapy preclinical mode.

  • Research Products

    (4 results)

All 2012 2011 2010

All Journal Article (4 results) (of which Peer Reviewed: 4 results)

  • [Journal Article] Lentivector transduction improves outcomes over transplantation of human HSCs alone in NOD/SCID/Fabry mice2012

    • Author(s)
      Pacienza N, Yoshimitsu M, Mizue N, Au BCY, Wang JCM, Fan X, Takenaka T, Medin JA
    • Journal Title

      Molecular Therapy

      Volume: 20 Pages: 1454-1461

    • Peer Reviewed
  • [Journal Article] Sequencing and characterization of the porcine alpha-galactosidase A gene: towards the generation of a porcine model for Fabry disease2011

    • Author(s)
      Yoshimitsu M, Higuchi K, Fan X, Takao S, Medin JA, Tei C, Takenaka T
    • Journal Title

      Mol Biol Rep

      Volume: 38 Pages: 3145-3152

    • Peer Reviewed
  • [Journal Article] Identification of novel mutations in the α-galactosidase A gene in patients with Fabry disease: Pitfalls of mutation analyses in patients with low α-galactosidase A activity2011

    • Author(s)
      Yoshimitsu M, Higuchi K, Miyata M, Devine S, Mattman A, Sirrs S, Medin JA, Tei C, Takenaka T
    • Journal Title

      Journal of Cardiology

      Volume: 57 Pages: 345-353

    • Peer Reviewed
  • [Journal Article] Alpha-galactosidase A-Tat fusion enhances storage reduction in hearts and kidneys of fabry mice2010

    • Author(s)
      Higuchi K, Yoshimitsu M, Fan X, Guo X, Rasaiah VI, Yen J, Tei C, Takenaka T
    • Journal Title

      Molecular Medicine

      Volume: 16 Pages: 216-221

    • Peer Reviewed

URL: 

Published: 2014-08-29  

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