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Homologous recombination-mediated gene correction for Adenosine deaminase deficiency

Research Project

Project/Area Number 23791147
Research Category

Grant-in-Aid for Young Scientists (B)

Allocation TypeMulti-year Fund
Research Field Pediatrics
Research InstitutionTohoku University

Principal Investigator

UCHIYAMA Toru  東北大学, 病院, 助教 (10436107)

Project Period (FY) 2011 – 2012
Project Status Completed (Fiscal Year 2012)
Budget Amount *help
¥4,030,000 (Direct Cost: ¥3,100,000、Indirect Cost: ¥930,000)
Fiscal Year 2012: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
Fiscal Year 2011: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
Keywords原発性免疫不全症 / 遺伝子治療 / 相同組換え / iPS細胞 / 造血幹細胞 / 相同組み替え / ADA欠損症
Research Abstract

iPS cells were derived from skin fibroblasts of ADA deficiency patients. While AAV-mediated gene targeting for the homologous recombination, followed by the selection with deoxyadenosine, was performed, corrected clones were not obtained. iPS cells showed the differentiation into T and B lymphocytes in the culture with OP9 stromal cells. This system is thought to be useful for the functional analysis of corrected ADA gene in iPS cells.

Report

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

    (19 results)

All 2013 2012 2011 Other

All Journal Article (8 results) (of which Peer Reviewed: 8 results) Presentation (11 results)

  • [Journal Article] Interstitial lung disease in two brothers with novel compound heterozygous ABCA3 mutations2013

    • Author(s)
      Kitazawa H, Moriya K, Niizuma H, Kawano K, Saito-Nanjo Y, Uchiyama T, Rikiishi T, Sasahara Y, Sakamoto O, Setoguchi Y, Kure S
    • Journal Title

      Eur J Pediatr

      Volume: 172(7) Issue: 7 Pages: 953-7

    • DOI

      10.1007/s00431-013-1977-8

    • Related Report
      2012 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Development of a Multi-Step Leukemogenesis Model of MLL-Rearranged Leukemia Using Humanized Mice2012

    • Author(s)
      Moriya K, Suzuki M, Watanabe Y, Takahashi T, Aoki Y, Uchiyama T, Kumaki S, Sasahara Y, Minegishi M, Kure S, Tsuchiya S, Sugamuta K, Ishii N.
    • Journal Title

      PLos One

      Volume: 7(6)

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Foamy Virus Vector-mediated Gene Correction of a Mouse Model of Wiskott-Aldrich Syndrome2012

    • Author(s)
      Uchiyama T, Adriani M, Jagadeesh GJ, Paine A, Candotti F
    • Journal Title

      Mol. Ther

      Volume: 20(6) Issue: 6 Pages: 1270-1279

    • DOI

      10.1038/mt.2011.282

    • Related Report
      2012 Annual Research Report 2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Successful cord blood transplantation with reduced-intensity conditioning for childhood cerebral X-linked adrenoleukodystrophy at advanced and early stages2012

    • Author(s)
      Niizuma H, Uematsu M, Sakamoto O, Uchiyama T, Horino S, Onuma M, Matsuhashi T, Rikiishi T, Sasahara Y, Minegishi M, Tsuchiya S.
    • Journal Title

      Pediatr Transplant

      Volume: 16(2) Issue: 2

    • DOI

      10.1111/j.1399-3046.2011.01539.x

    • Related Report
      2012 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Development of a multi-step leukemogenesis model of MLL-rearranged leukemia using humanized mice2012

    • Author(s)
      Moriya K, Suzuki M, Watanabe Y, Takahashi T, Aoki Y, Uchiyama T, Kumaki S, Sasahara Y, Minegishi M, Kure S, Tsuchiya S, Sugamura K, and Ishii N.
    • Journal Title

      PLoS ONE

      Volume: 7(6) Issue: 6 Pages: e37892-e37892

    • DOI

      10.1371/journal.pone.0037892

    • Related Report
      2012 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Defective inhibition of B-cell proliferation by Wiskott-Aldrich syndrome protein-deficient regulatory T cells2011

    • Author(s)
      Adriani M, Jones KA, Uchiyama T, Kirby MR, Silvin C, Anderson SM, Candotti F
    • Journal Title

      Blood

      Volume: 117(24) Issue: 24 Pages: 6608-6611

    • DOI

      10.1182/blood-2010-12-322834

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Interstitial lung disease in two brothers with novel compound heterozygous ABCA3 mutations

    • Author(s)
      Kitazawa H., Moriya K., Niizuma H., Kawano K., Saito-Nanjo Y., Uchiyama T., Rikiishi T., Sasahara Y., Sakamoto O., Setoguchi Y., Kure S
    • Journal Title

      Eur J Pediatr

      Volume: (In press)

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Successful cord blood transplantation with reduced-intensity conditioning for childhood cerebral X-linked adrenoleukodystorophy at advanced and early stages

    • Author(s)
      Niizuma H, Uematsu M, Sakamoto O, Uchiyama T, Horino S, Onuma M, Matsuhashi T, Rikiishi T, Sasahara Y, Minegishi M, Tsuchiya S.
    • Journal Title

      Pediatr. Transplant

      Volume: 16(2)

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Presentation] Development of Foamy Virus Vector System for Primaryimmunodeficiencies2012

    • Author(s)
      Toru Uchiyama 、Satoshi Horino 、Yoji Sasahara、Naoto Ishii、Fabio Candotti
    • Organizer
      第18回日本遺伝子治療学会
    • Place of Presentation
      熊本
    • Year and Date
      2012-06-28
    • Related Report
      2012 Final Research Report
  • [Presentation] Spontaneous reversionを伴う原発性免疫不全症2症例における長期的臨床経過の検討2012

    • Author(s)
      笹原洋二、佐藤美季、斉藤由佳、小沼正栄、入江正寛、内山徹、力石健、呉繁夫
    • Organizer
      第115回日本小児科学会学術集会
    • Place of Presentation
      福岡
    • Year and Date
      2012-04-20
    • Related Report
      2012 Final Research Report
  • [Presentation] 骨髄非破壊的前処置にて同種骨髄移植を施行した慢性活動性EBV感染症4例の臨床的検討2012

    • Author(s)
      笹原洋二、渡辺祐子、小沼正栄、新妻秀剛、内山徹、力石健、峯岸政好、久間木悟、土屋滋
    • Organizer
      第34回日本造血幹細胞移植学会学術集会
    • Place of Presentation
      大阪
    • Year and Date
      2012-02-24
    • Related Report
      2012 Final Research Report
  • [Presentation] RISTを施行した当科年長児および成人期WAS/XLT3症例の臨床的検討2012

    • Author(s)
      笹原洋二、力石健、北沢博、内山徹、森尾友宏、峯岸政好、久間木悟、土屋滋
    • Organizer
      第5回日本免疫不全症研究会
    • Place of Presentation
      東京
    • Year and Date
      2012-01-21
    • Related Report
      2012 Final Research Report
  • [Presentation] リツキシマブが有効であった乳児自己免疫性溶血性貧血の1例2012

    • Author(s)
      森谷邦彦、松橋徹朗、小沼正栄、内山徹、力石健、浅田洋司、鈴木潤、笹原洋二、呉繁夫
    • Organizer
      第54回小児血液・がん学会学術集会
    • Place of Presentation
      横浜
    • Related Report
      2012 Final Research Report
  • [Presentation] Development of Foamy virus vectors for primary immunodeficiencies.2012

    • Author(s)
      内山徹
    • Organizer
      第18回日本遺伝子治療学会
    • Place of Presentation
      熊本
    • Related Report
      2012 Annual Research Report
  • [Presentation] Spontaneous reversionを伴う原発性免疫不全症2症例における長期的臨床経過の検討2012

    • Author(s)
      笹原洋二
    • Organizer
      第115回日本小児科学会学術集会
    • Place of Presentation
      福岡
    • Related Report
      2012 Annual Research Report
  • [Presentation] リツキシマブが有効であった乳児自己免疫性溶血性貧血の一例2012

    • Author(s)
      森谷邦彦
    • Organizer
      第54回小児血液がん学会
    • Place of Presentation
      横浜
    • Related Report
      2012 Annual Research Report
  • [Presentation] SKY法が複雑型染色体異常の同定に有用であった急性白血病の2例2011

    • Author(s)
      片山紗乙莉、小沼正栄、内山徹、新妻秀剛、力石健、笹原洋二、呉繁夫
    • Organizer
      第53回日本小児血液・がん学会学術集会
    • Place of Presentation
      前橋
    • Year and Date
      2011-11-25
    • Related Report
      2012 Final Research Report
  • [Presentation] フォーミーウイルスベクターによるWiskott-Aldrich症候群への遺伝子治療2011

    • Author(s)
      内山徹、Fabio Candotti、土屋滋
    • Organizer
      第114回日本小児科学会学術集会
    • Place of Presentation
      東京
    • Year and Date
      2011-08-12
    • Related Report
      2012 Final Research Report
  • [Presentation] The progress of stem cell gene therapy for primary immunodefici- ency2011

    • Author(s)
      Toru Uchiyama
    • Organizer
      第17回日本遺伝子治療学会学術集会、シンポジウム
    • Place of Presentation
      福岡
    • Year and Date
      2011-07-15
    • Related Report
      2012 Final Research Report

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

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