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Generation of gene therapy method for ataxia-telangiectasia using a human artificial chromosome

Research Project

Project/Area Number 19790246
Research Category

Grant-in-Aid for Young Scientists (B)

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

Principal Investigator

KAZUKI Yasuhiro  Tottori University, 大学院・医学系研究科, 助教 (90403401)

Project Period (FY) 2007 – 2009
Project Status Completed (Fiscal Year 2009)
Budget Amount *help
¥3,640,000 (Direct Cost: ¥3,100,000、Indirect Cost: ¥540,000)
Fiscal Year 2009: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2008: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2007: ¥1,300,000 (Direct Cost: ¥1,300,000)
Keywordsヒト人工染色体 / 遺伝子治療 / 染色体工学 / 毛細血管拡張性運動失調症 / ES細胞 / 再生医療 / 遣伝子治療
Research Abstract

To generate gene therapy vector for ataxia telangiectasia (AT), we constructed novel human artificial chromosome (HAC) containing ATM gene, which is mutated in ataxia telangiectasia (AT) patients. The ATM-HAC were transferred into ATM-deficient cell line derived from a AT patient. The ATM-deficient phenotypes of gene regulation and radiation sensitivity were complemented by introducing the ATM-HAC. Furthermore, the ATM-HAC was stably maintained in the cell lines. Thus, the ATM-HAC vector will be useful for gene therapy of AT.

Report

(4 results)
  • 2009 Annual Research Report   Final Research Report ( PDF )
  • 2008 Annual Research Report
  • 2007 Annual Research Report
  • Research Products

    (22 results)

All 2010 2009 2008 2007

All Journal Article (12 results) (of which Peer Reviewed: 10 results) Presentation (8 results) Patent(Industrial Property Rights) (2 results) (of which Overseas: 1 results)

  • [Journal Article] Complete genetic correction of iPS cells from Duchenne muscular dystrophy.2010

    • 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., Oshimura M.
    • Journal Title

      Mol. Therapy. Volume18,number2

      Pages: 386-93

    • Related Report
      2009 Final Research Report
    • Peer Reviewed
  • [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 Vol.9,No.3

      Pages: 37-40

    • Related Report
      2009 Final Research Report
  • [Journal Article] A highly stable and non-integrated human artificial chromosome (HAC) containing the 2. 4Mb entire human dystrophin gene.2009

    • Author(s)
      Hoshiya H., Kazuki Y., Abe S., Takiguchi M., Kajitani N., Watanabe Y., Yoshino T., Shirayoshi Y., Higaki K., Messina G., Cossu G., Oshimura M.
    • Journal Title

      Mol. Therapy. Volume17,number2

      Pages: 309-17

    • Related Report
      2009 Final Research Report
    • Peer Reviewed
  • [Journal Article] Enhanced apoptosis during early neuronal differentiation in mouse ES cells with autosomal imbalance.2009

    • Author(s)
      Kai Y., Wang CC., Kishigami S., Kazuki Y., Abe S., Takiguchi M., Shirayoshi Y., Inoue T., Ito H., Wakayama T., Oshimura M.
    • Journal Title

      Cell Res. Volume19,number2

      Pages: 247-58

    • Related Report
      2009 Final Research Report
    • Peer Reviewed
  • [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
  • [Journal Article] A highly stable and non-integrated human artificial chromosome(HAC) containing the 2.4Mb entire human dystrophin gene2009

    • Author(s)
      Hoshiya H.
    • Journal Title

      Mol. Therapy 17

      Pages: 309-317

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Enhanced apoptosis during early neuronal differentiation in mouse ES cells with autosomal imbalance2009

    • Author(s)
      Kai Y.
    • Journal Title

      Cell Res 19

      Pages: 247-258

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Telomerase-mediated life-span extension of human primary fibroblasts by human artificial chromosome (HAC) vector.2008

    • Author(s)
      Shitara S, Kakeda M, Nagata K, Hiratsuka M, Sano A, Osawa K, Okazaki A, Katoh M, Kazuki Y, Oshimura M, Tomizuka K.
    • Journal Title

      Biochem Biophys Res Commun. Volume369,number3

      Pages: 807-11

    • Related Report
      2009 Final Research Report
    • Peer Reviewed
  • [Journal Article] Correction of a genetic defect in multipotent germline stem cells using a human artificial chromosome.2008

    • Author(s)
      Kazuki Y, Hoshiy H, Kai Y, Abe S, Takiguch M, Osaki M, Kawazoe S, Katoh M, Kanatsu-Shinohara M, Inoue K, Kajitani N, Yoshino T, Shirayoshi S, Ogura A, Shinohara T, Barrett JC, Oshimura M.
    • Journal Title

      Gene therapy Volume15,number8

      Pages: 617-24

    • Related Report
      2009 Final Research Report
    • Peer Reviewed
  • [Journal Article] Telomerase-mediated life-span extension of primary fibroblasts by human artificial chromosome (HAC) vector.2008

    • Author(s)
      Shitara S
    • Journal Title

      Biochem Biophys Res Commun. 369

      Pages: 807-811

    • Related Report
      2007 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Correction of a genetic defect in multipotent germline stem cell using a human artificial chromosome.2008

    • Author(s)
      Kazuki Y
    • Journal Title

      Gene Ther. 15

      Pages: 617-624

    • Related Report
      2007 Annual Research Report
    • Peer Reviewed
  • [Presentation] 染色体工学技術を用いたヒト型モデル動物の作製と応用2009

    • Author(s)
      香月康宏
    • Organizer
      第129回日本薬学会年会
    • Place of Presentation
      京都
    • Year and Date
      2009-03-26
    • Related Report
      2008 Annual Research Report
  • [Presentation] ヒト型P450薬物代謝酵素発現マウス2009

    • Author(s)
      香月康宏, 小林カオル, 千葉寛, 押村光雄
    • Organizer
      第56回日本実験動物学会総会
    • Place of Presentation
      埼玉
    • Related Report
      2009 Annual Research Report 2009 Final Research Report
  • [Presentation] 染色体工学技術を用いたヒト型モデル動物の作製と応用2009

    • Author(s)
      香月康宏, 押村光雄
    • Organizer
      第129回日本薬物学会年会
    • Place of Presentation
      京都
    • Related Report
      2009 Final Research Report
  • [Presentation] A novel human artificial chromosome (HAC) for gene therapy and animal transgenesisASGT 11^<th> annual meeting2008

    • Author(s)
      Kazuki Y
    • Organizer
      ASGT 11^<th> annual meeting
    • Place of Presentation
      USA, Boston
    • Year and Date
      2008-05-30
    • Related Report
      2008 Annual Research Report
  • [Presentation] A novel human artificial chromosome (HAC) for gene therapy and animal transgenesis2008

    • Author(s)
      Kazuki Y., Hoshiya H., Abe S., Takiguchi M., Iida Y., Watanabe Y., Osaki M., Katoh M., Kajitani N., Yoshino T., Shirayoshi Y., Hiratsuka M., Kurimasa K., Oshimura M.
    • Organizer
      ASGT 11th annual meeting
    • Place of Presentation
      U.S.A.
    • Related Report
      2009 Final Research Report
  • [Presentation] 遺伝子治療に向けた新規遺伝子治療用ヒト人工染色体ベクターの開発2008

    • Author(s)
      香月康宏, 星谷英寿, 甲斐義輝, 阿部智志, 滝口正人, 飯田雄一, 渡辺義徳, 尾崎克彦, 梶谷尚世, 吉野とう子, 白吉安昭, 富松望, 平塚正治, 栗政明弘, 押村光雄
    • Organizer
      第7回日本再生医療学会総会
    • Place of Presentation
      名古屋
    • Related Report
      2009 Final Research Report 2007 Annual Research Report
  • [Presentation] Genetic restoration of ataxia telangiectasia cells using a human artificial chromosome mediated- genomic cloning system2007

    • Author(s)
      Kazuki Y
    • Organizer
      ASGT 10th annual meeting
    • Place of Presentation
      USA,Seattle
    • Year and Date
      2007-06-01
    • Related Report
      2007 Annual Research Report
  • [Presentation] Genetic restoration of ataxia telangiectasia cells using a human artificial chromosome mediatedgenomic cloning system2007

    • Author(s)
      Kazuki Y., Hoshiya, H., Kai Y., Abe S., Takiguchi M., Osaki M., Yuichi I., Mizutani E., Kajitani N., Yoshino T., Shirayoshi Y., Tomimatsu N., Hratsuka M., Kurimasa A., Wakayama T., Oshimura, M.
    • Organizer
      ASGT 10th annual meeting
    • Place of Presentation
      U.S.A.
    • Related Report
      2009 Final Research Report
  • [Patent(Industrial Property Rights)] ヒト人工染色体(HAC)ベクター及びヒト人工染色体(HAC)を有するヒト細胞医薬2007

    • Inventor(s)
      掛田実, 富塚一磨, 押村光雄, 香月康宏
    • Industrial Property Rights Holder
      キリンファーマ株式会社
    • Filing Date
      2007-07-06
    • Related Report
      2009 Final Research Report
  • [Patent(Industrial Property Rights)] ヒト人工染色体(HAC)ベクター及びヒト人工染色体(HAC)を有するヒト細胞医薬2007

    • Inventor(s)
      掛田実、富塚一磨、押村光雄、香月康宏
    • Industrial Property Rights Holder
      キリンファーマ株式会社
    • Filing Date
      2007-07-06
    • Related Report
      2007 Annual Research Report
    • Overseas

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Published: 2007-04-01   Modified: 2016-04-21  

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