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Development of a therapeutic genome editing for hyper-IgE syndrome

Research Project

Project/Area Number 15K15392
Research Category

Grant-in-Aid for Challenging Exploratory Research

Allocation TypeMulti-year Fund
Research Field Pediatrics
Research InstitutionThe University of Tokushima

Principal Investigator

MINEGISHI Yoshiyuki  徳島大学, 疾患プロテオゲノム研究センター, 教授 (10343154)

Project Period (FY) 2015-04-01 – 2016-03-31
Project Status Completed (Fiscal Year 2015)
Budget Amount *help
¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2015: ¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Keywords免疫学 / ゲノム編集 / 小児科学 / 免疫不全症 / STAT3
Outline of Final Research Achievements

Hyper-IgE syndrome is characterized by the extremely high serum IgE levels, severe atopic dermatitis, and recurrent staphylococcal infections to the skin and lung. This disorder is caused by a substitution of a single nucleotide of a single allele of STAT3 gene. To treat this disorder with genome editing, we need to establish a CRISPER/Cas9, which could discriminate a difference of the single nucleotide. To this end, we established a reporter assay, specifically detect double strand break by CRISPER/Cas9. With this model system, we successfully identified a CRISPR/Cas9, which specifically cleave mutant STAT3 allele, but not cleave wild-type STAT3 allele.

Report

(2 results)
  • 2015 Annual Research Report   Final Research Report ( PDF )
  • Research Products

    (10 results)

All 2016 2015

All Journal Article (7 results) (of which Int'l Joint Research: 2 results,  Peer Reviewed: 2 results,  Open Access: 2 results) Presentation (3 results) (of which Int'l Joint Research: 3 results)

  • [Journal Article] Human TYK2 deficiency: Mycobacterial and viral infections without hyper-IgE syndrome.2015

    • Author(s)
      Kreins A.Y, Ciancanelli M.J, Boisson-Dupuis S, et al.
    • Journal Title

      J Exp Med.

      Volume: 212 Issue: 10 Pages: 1641-1662

    • DOI

      10.1084/jem.20140280

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Monogenic mutations differentially affect the quantity and quality of T follicular helper cells in patients with human primary immunodeficiencies.2015

    • Author(s)
      Ma CS, Wong N, Tangye SG, et al.
    • Journal Title

      J Allergy Clin Immunol

      Volume: 136 Issue: 4 Pages: 993-1006

    • DOI

      10.1016/j.jaci.2015.05.036

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] 高IgE症候群2015

    • Author(s)
      峯岸克行
    • Journal Title

      臨床免疫アレルギー科

      Volume: 63 Pages: 251-253

    • Related Report
      2015 Annual Research Report
  • [Journal Article] AD-HIES (Job’s症候群)2015

    • Author(s)
      峯岸克行
    • Journal Title

      日本臨床 免疫症候群III

      Volume: 34 Pages: 235-237

    • Related Report
      2015 Annual Research Report
  • [Journal Article] Comel-Netherton症候群2015

    • Author(s)
      峯岸克行
    • Journal Title

      日本臨床 免疫症候群III

      Volume: 34 Pages: 238-239

    • Related Report
      2015 Annual Research Report
  • [Journal Article] PGM3欠損症2015

    • Author(s)
      峯岸克行
    • Journal Title

      日本臨床 免疫症候群III

      Volume: 34 Pages: 240-241

    • Related Report
      2015 Annual Research Report
  • [Journal Article] 高IgE症候群 (Job症候群)2015

    • Author(s)
      峯岸克行
    • Journal Title

      小児科診療

      Volume: 79 Pages: 352-352

    • Related Report
      2015 Annual Research Report
  • [Presentation] Molecular pathogenesis of hyper-IgE syndrome2016

    • Author(s)
      Minegishi Y
    • Organizer
      The 5th Bizan Immunology Symposium
    • Place of Presentation
      Tokushima University (徳島県・徳島市)
    • Year and Date
      2016-03-03
    • Related Report
      2015 Annual Research Report
    • Int'l Joint Research
  • [Presentation] Dysregulationof T cell dependent antibody response in a murine model of hyper IgE syndrome2015

    • Author(s)
      Nishikawa Y, Wada T, Minegishi Y
    • Organizer
      The 44th annual meeting of the Japanese society for Immunology
    • Place of Presentation
      Sapporo Convention Center (北海道・札幌市)
    • Year and Date
      2015-11-18
    • Related Report
      2015 Annual Research Report
    • Int'l Joint Research
  • [Presentation] Hyper-IgE syndrome model mice exhibit the susceptibility to Staphyloccocus aureus infection2015

    • Author(s)
      Wada T, Nishikawa Y, Minegishi Y
    • Organizer
      The 44th annual meeting of the Japanese society for Immunology
    • Place of Presentation
      Sapporo Convention Center (北海道・札幌市)
    • Year and Date
      2015-11-18
    • Related Report
      2015 Annual Research Report
    • Int'l Joint Research

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Published: 2015-04-16   Modified: 2017-05-10  

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