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Gene therapy of Duchenne muscular dystrophy using own stem cells and human artificial chromosome

Research Project

Project/Area Number 21249022
Research Category

Grant-in-Aid for Scientific Research (A)

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

Principal Investigator

OSHIMURA Mitsuo  鳥取大学, 大学院・医学系・研究科, 教授 (20111619)

Co-Investigator(Kenkyū-buntansha) OSAKI Mitsuhiko  鳥取大学, 医学部, 准教授 (40325006)
KAZUKI Yasuhiro  鳥取大学, 大学院・医学系・研究科, 助教 (90403401)
Project Period (FY) 2009 – 2011
Project Status Completed (Fiscal Year 2011)
Budget Amount *help
¥46,410,000 (Direct Cost: ¥35,700,000、Indirect Cost: ¥10,710,000)
Fiscal Year 2011: ¥14,950,000 (Direct Cost: ¥11,500,000、Indirect Cost: ¥3,450,000)
Fiscal Year 2010: ¥14,950,000 (Direct Cost: ¥11,500,000、Indirect Cost: ¥3,450,000)
Fiscal Year 2009: ¥16,510,000 (Direct Cost: ¥12,700,000、Indirect Cost: ¥3,810,000)
Keywords遺伝子治療学 / 染色体工学 / ヒト人工染色体 / 遺伝子治療 / 筋ジストロフィー / 間葉系幹細胞 / mesoangioblast細胞
Research Abstract

Toward gene therapy of Duchenne muscular dystrophy(DMD), we transferred the human artificial chromosome with 2. 4 Mb genomic dystrophin gene(DYS-HAC) to the DMD mouse model(mdx)-derived mesoangioblasts, then the corrected mesoangioblasts were transplanted to mdx-mice. As a result, we succeeded in the long term-amelioration of the dystrophic phenotype in the mdx mice. Thus, a combination of HAC-mediated gene replacement and transplantation with mesoangioblasts will be useful for gene therapy of DMD.

Report

(4 results)
  • 2011 Annual Research Report   Final Research Report ( PDF )
  • 2010 Annual Research Report
  • 2009 Annual Research Report
  • Research Products

    (36 results)

All 2012 2011 2010 2009 Other

All Journal Article (14 results) (of which Peer Reviewed: 6 results) Presentation (18 results) Remarks (4 results)

  • [Journal Article] Human Artificial Chromosomes for Gene Delivery and the Development of Animal Models2011

    • Author(s)
      Kazuki Y, Oshimura M.
    • Journal Title

      Mol Ther

      Volume: Volume 19, number 9 Issue: 9 Pages: 1591-601

    • DOI

      10.1038/mt.2011.136

    • Related Report
      2011 Annual Research Report 2011 Final Research Report
    • Peer Reviewed
  • [Journal Article] Stem cell-mediated transfer of a human artificial chromosome ameliorates muscular dystrophy2011

    • Author(s)
      Tedesco FS, Hoshiya H, D' Antona G, Gerli MF, Messina G, Antonini S, Tonlorenzi R, Benedetti S, Berghella L, Torrente Y, Kazuki Y, Bottinelli R, Oshimura M, Cossu G.
    • Journal Title

      Sci Transl Med

      Volume: Volume 3, number 96 Issue: 96 Pages: 96-78

    • DOI

      10.1126/scitranslmed.3002342

    • Related Report
      2011 Annual Research Report 2011 Final Research Report
    • Peer Reviewed
  • [Journal Article] ヒト人工染色体ベクターによる遺伝子細胞治療へ向けて2011

    • Author(s)
      香月康宏、押村光雄
    • Journal Title

      日本臨床

      Volume: Vol.69, No.12 Pages: 2142-7

    • Related Report
      2011 Final Research Report
  • [Journal Article] ヒト人工染色体ベクターによる遺伝子細胞治療へ向けて2011

    • Author(s)
      香月康宏
    • Journal Title

      日本臨床

      Volume: 69 Pages: 2142-2147

    • Related Report
      2011 Annual Research Report
  • [Journal Article] Refined human artificial chromosome vectors for gene therapy and animal transgenesis.2011

    • Author(s)
      Kazuki Y.
    • Journal Title

      Gene Therapy

      Volume: 18 Pages: 384-393

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] ヒト人工染色体ベクターの利点とその応用2010

    • Author(s)
      山口繁幸、大林徹也、香月康宏、押村光雄
    • Journal Title

      生化学

      Volume: Vol.82, No.9 Pages: 846-852

    • NAID

      10027502606

    • Related Report
      2011 Final Research Report
  • [Journal Article] Refined human artificial chromosome vectors for gene therapy and animal transgenesis2010

    • Author(s)
      Kazuki Y, Hoshiya H, Takiguchi M, Abe S, Iida Y, Osaki M, Katoh M, Hiratsuka M, Shirayoshi Y, Hiramatsu K, Ueno E, Kajitani N, Yoshino T, Kazuki K, Ishihara C, Takehara S, Tsuji S, Ejima F, Toyoda A, Sakaki Y, Larionov V, Kouprina N and Oshimura M
    • Journal Title

      Gene therapy

      Volume: Volume 18, number 4 Issue: 4 Pages: 384-93

    • DOI

      10.1038/gt.2010.147

    • Related Report
      2011 Final Research Report
    • Peer Reviewed
  • [Journal Article] 筋ジストロフィー患者由来のiPS細胞における遺伝子修復~ヒト人工染色体ベクターによる新たな遺伝子治療戦略の可能性2010

    • Author(s)
      香月康宏、押村光雄
    • Journal Title

      Medical Bio

      Volume: Vol.7, No.2 Pages: 58-63

    • Related Report
      2011 Final Research Report
  • [Journal Article] Complete genetic correction of iPS cells from Duchenne muscular dystrophy2010

    • Author(s)
      Kazuki Y., Hiratsuka M., Takiguchi M., Osaki M., Kajitani N., Hoshiya H., Hiramatsu K., Yoshino T., Kazuki K., Ishihara C., Takehara S., Higaki K., Nakagawa M., Takahashi K., Yamanaka S. and Oshimura M.
    • Journal Title

      Mol. Therapy

      Volume: Volume18, number 2 Issue: 2 Pages: 386-93

    • DOI

      10.1038/mt.2009.274

    • Related Report
      2011 Final Research Report
    • Peer Reviewed
  • [Journal Article] ヒト人工染色体ベクターの利点とその応用2010

    • Author(s)
      山口繁幸
    • Journal Title

      生化学

      Volume: 82 Pages: 846-852

    • Related Report
      2010 Annual Research Report
  • [Journal Article] 筋ジストロフィー患者由来のiPS細胞における遺伝子修復~ヒト人工染色体ベクターによる新たな遺伝子治療戦略の可能性2010

    • Author(s)
      香月康宏
    • Journal Title

      Medical Bio 7

      Pages: 58-63

    • Related Report
      2009 Annual Research Report
  • [Journal Article] 幹細胞のゲノム操作:ヒト人工染色体ベクターの開発2009

    • Author(s)
      香月康宏、押村光雄
    • Journal Title

      ゲノム医学

      Volume: Vol.9, No.3 Pages: 37-40

    • Related Report
      2011 Final Research Report
  • [Journal Article] Complete genetic correction of iPS cells from Duchenne muscular dystrophy2009

    • Author(s)
      Kazuki Y.
    • Journal Title

      Mol.Therapy 18

      Pages: 386-393

    • Related Report
      2009 Annual Research Report
    • Peer Reviewed
  • [Journal Article] ヒト人工染色体ベクターの開発2009

    • Author(s)
      香月康宏
    • Journal Title

      ゲノム医学 9

      Pages: 37-40

    • Related Report
      2009 Annual Research Report
  • [Presentation] 遺伝子発現ベクターとしての人工染色体2012

    • Author(s)
      押村光雄
    • Organizer
      再生医療の実現化プロジェクト」ヒト多能性幹細胞の培養・解析の標準化レクチャーシリーズ第6回「幹細胞の遺伝子操作の実践(つぼ)
    • Place of Presentation
      理化学研究所(兵庫県)
    • Year and Date
      2012-02-02
    • Related Report
      2011 Final Research Report
  • [Presentation] 遺伝子発現ベクターとしての人工染色体2012

    • Author(s)
      押村光雄
    • Organizer
      文部科学省「再生医療の実現化プロジェクト」ヒト多能性幹細胞の培養・解析の標準化レクチャーシリーズ第6回「幹細胞の遺伝子操作の実践(つぼ)」
    • Place of Presentation
      理化学研究所(兵庫県)(招待講演)
    • Year and Date
      2012-02-02
    • Related Report
      2011 Annual Research Report
  • [Presentation] Human artificial chromosomes for gene delivery and stable expression2012

    • Author(s)
      Oshimura M
    • Organizer
      11th Annual PEP TALK
    • Place of Presentation
      SanDiego(Califolnia)
    • Year and Date
      2012-01-09
    • Related Report
      2011 Final Research Report
  • [Presentation] Human artificial chromosomes for gene delivery and stable expression2012

    • Author(s)
      Oshimura M
    • Organizer
      11^<th> Annual PEP TALK
    • Place of Presentation
      San Diego (Califolnia)
    • Year and Date
      2012-01-09
    • Related Report
      2011 Annual Research Report
  • [Presentation] Novel human artificial chromosome vector for gene delivery2011

    • Author(s)
      Kazuki Y
    • Organizer
      第34回分生生物学会年会
    • Place of Presentation
      パシフィコ横浜(神奈川県)
    • Year and Date
      2011-12-13
    • Related Report
      2011 Annual Research Report 2011 Final Research Report
  • [Presentation] ヒト人工染色体を用いた遺伝子・再生医療への挑戦2011

    • Author(s)
      押村光雄
    • Organizer
      文部科学省iPS細胞等研究ネットワーク第3回合同シンポジウム
    • Place of Presentation
      国立京都国際会館(京都府)
    • Year and Date
      2011-11-19
    • Related Report
      2011 Final Research Report
  • [Presentation] ヒト人工染色体を用いた遺伝子・再生医療への挑戦2011

    • Author(s)
      押村光雄
    • Organizer
      文部科学省iPS細胞等研究ネットワーク第3回合同シンポジウム
    • Place of Presentation
      国立京都国際会館(京都府)(招待講演)
    • Year and Date
      2011-11-19
    • Related Report
      2011 Annual Research Report
  • [Presentation] Human artificial chromosomes for gene delivery2011

    • Author(s)
      Oshimura M
    • Organizer
      World stem cell summit
    • Place of Presentation
      Pasadena(Califolnia)
    • Year and Date
      2011-10-03
    • Related Report
      2011 Final Research Report
  • [Presentation] Human artificial chromosomes for gene delivery2011

    • Author(s)
      Oshimura M
    • Organizer
      World stem cell summit
    • Place of Presentation
      Pasadena (Califolnia)
    • Year and Date
      2011-10-03
    • Related Report
      2011 Annual Research Report
  • [Presentation] 遺伝子再生医療・ヒト型モデル動物作製のためのヒト人工染色体ベクター(HAC)システムの開発2011

    • Author(s)
      香月康宏
    • Organizer
      第10回日本再生医療学会総会
    • Place of Presentation
      京王プラザホテル(東京)
    • Year and Date
      2011-03-02
    • Related Report
      2011 Final Research Report 2010 Annual Research Report
  • [Presentation] Human artificial chromosome and its medical and pharmaceutical applications2010

    • Author(s)
      押村光雄
    • Organizer
      The 4th Asian Chromosome Colloquium(ACC4) Plenary Lectures
    • Place of Presentation
      Fragrant Hill Empark Hotel(北京・中国)
    • Year and Date
      2010-10-12
    • Related Report
      2011 Final Research Report
  • [Presentation] Human artificial chromosome and its medical and pharmaceutical applications2010

    • Author(s)
      押村光雄
    • Organizer
      The 4^<th> Asian Chromosome Colloquium (ACC4) Plenary Lectures
    • Place of Presentation
      Fragrant Hill Empark Hotel(北京・中国)
    • Year and Date
      2010-10-12
    • Related Report
      2010 Annual Research Report
  • [Presentation] 人工染色体を用いた遺伝子/再生医療2010

    • Author(s)
      押村光雄
    • Organizer
      第180回川崎医学会講演会
    • Place of Presentation
      川崎医科大学(倉敷)
    • Year and Date
      2010-05-12
    • Related Report
      2011 Final Research Report
  • [Presentation] 人工染色体を用いた遺伝子/再生医療2010

    • Author(s)
      押村光雄
    • Organizer
      第180回川崎医学会講演会
    • Place of Presentation
      川崎医科大学(倉敷)(招待講演)
    • Year and Date
      2010-05-12
    • Related Report
      2010 Annual Research Report
  • [Presentation] ジストロフィン2. 4Mb搭載ヒト人工染色体による筋ジストロフィー由来iPS細胞の遺伝子完全修復2010

    • Author(s)
      香月康宏
    • Organizer
      第9回日本再生医療学会総会
    • Place of Presentation
      広島
    • Year and Date
      2010-03-18
    • Related Report
      2011 Final Research Report
  • [Presentation] ジストロフィン2.4Mb搭載ヒト人工染色体による筋ジストロフィー由来iPS細胞の遺伝子完全修復2010

    • Author(s)
      香月康宏
    • Organizer
      第9回日本再生医療学会総会
    • Place of Presentation
      広島
    • Year and Date
      2010-03-18
    • Related Report
      2009 Annual Research Report
  • [Presentation] Correction of Duchenne muscular dystrophy in induced pluripotent stem cells using a human artificial chromosome2009

    • Author(s)
      Kazuki Y
    • Organizer
      ASHG 59th annual meeting
    • Place of Presentation
      USA、ハワイ
    • Year and Date
      2009-10-21
    • Related Report
      2011 Final Research Report
  • [Presentation] Correction of Duchenne muscular dystrophy in induced pluripotent stem cells using a human artificial chromosome2009

    • Author(s)
      Kazuki Y
    • Organizer
      ASHG 59^<th> annual meeting
    • Place of Presentation
      USA.ハワイ
    • Year and Date
      2009-10-21
    • Related Report
      2009 Annual Research Report
  • [Remarks] 鳥取大学ホームページ

    • URL

      http://www.tottori-u.ac.jp/dd.aspx?itemid=7273#moduleid10

    • Related Report
      2011 Final Research Report
  • [Remarks] 毎日新聞:筋ジス遺伝子治療へ前進。2011年8月20日

    • Related Report
      2011 Final Research Report
  • [Remarks] 読売新聞:科学Monday筋ジス進行押さえた. 2011年10月31日

    • Related Report
      2011 Final Research Report
  • [Remarks]

    • URL

      http://www.tottori-u.ac.jp/dd.aspx?itemid=7273#moduleid10

    • Related Report
      2011 Annual Research Report

URL: 

Published: 2009-04-01   Modified: 2016-04-21  

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