• Search Research Projects
  • Search Researchers
  • How to Use
  1. Back to previous page

Analysis of the gene therapy for epidermolysis bullosa using transgenic rescue experiments of the disease model mice

Research Project

Project/Area Number 20790781
Research Category

Grant-in-Aid for Young Scientists (B)

Allocation TypeSingle-year Grants
Research Field Dermatology
Research InstitutionHokkaido University

Principal Investigator

ITO Kei  Hokkaido University, 北海道大学病院, 助教 (20421977)

Project Period (FY) 2008 – 2009
Project Status Completed (Fiscal Year 2009)
Budget Amount *help
¥4,290,000 (Direct Cost: ¥3,300,000、Indirect Cost: ¥990,000)
Fiscal Year 2009: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2008: ¥2,600,000 (Direct Cost: ¥2,000,000、Indirect Cost: ¥600,000)
Keywords皮膚病理学 / 遺伝子治療
Research Abstract

Recessive dystrophic epidermolysis bullosa comprises a group of hereditary bullous disease caused by mutations in the type VII collagen gene (COL7A1) that is a major component of anchoring fibril. We have investigated anchoring fibril formation in the Col7a1 knockout mice that express human COL7A1 in epidermal keratinocytes or dermal fibroblasts, using transgenic rescue experiments. In both rescue mice, normal anchoring fibril formation was seen within basement membrane zone. These data indicate that we can select either keratinocytes or fibroblasts as target cells in case of gene therapy for epidermolysis bullosa. Furthermore, using same transgenic rescue experiments, we were able to generate surviving animal models of epidermolysis bullosa with mutated human COL7A1 gene. This model has great potential for future research into the pathomechanisms of epidermolysis bullosa and the development of gene therapies for epidermolysis bullosa patients.

Report

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

    (8 results)

All 2010 2009 2008

All Journal Article (6 results) (of which Peer Reviewed: 6 results) Presentation (2 results)

  • [Journal Article] Prevalent LIPH founder mutations lead to loss of P2Y5 activation ability of PA-PLA1alpha in autosomal recessive hypotrichosis2010

    • Author(s)
      Shinkuma S, Ito K
    • Journal Title

      Human Mutation Mar 8(In press)

    • Related Report
      2009 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Keratinocyte-/fibroblast-targeted rescue of Col7a1 disrupted mice and generation of an exact dystrophic epidermolysis bullosa model using a human COL7A1 mutation2009

    • Author(s)
      Kei Ito, Daisuke Sawamura, Maki Goto, Hideki Nakamura, Wataru Nishie, Kaori Sakai, Ken Natsuga, Satoru Shinkuma, Akihiko Shibaki, Jouni Uitto, Christopher P. Denton, Osamu Nakajima, Masashi Akiyama, Hiroshi Shimizu
    • Journal Title

      The American Journal of Pathology Vol175

      Pages: 2508-2517

    • Related Report
      2009 Final Research Report
    • Peer Reviewed
  • [Journal Article] Keratinocyte-/fibroblast-targeted rescue of Col7a1-disrupted mice and generation of an exact dystrophic epidermolysis bullosa model using ahuman COL7A1 mutation2009

    • Author(s)
      Ito K, Shimizu H
    • Journal Title

      American Journal of Pathology 175

      Pages: 2508-2517

    • Related Report
      2009 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Pemphigus foliaceus associated with oesophageal cancer2009

    • Author(s)
      Shinkuma S, Ito K
    • Journal Title

      Journal of European Academy of Dermatology and Venereology 23

      Pages: 473-474

    • Related Report
      2009 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Pemphigus foliaceus associated with oesophageal cancer2009

    • Author(s)
      Shinkuma S, Ito K
    • Journal Title

      Journal of European Academy of Dermatology and Venereology 23(4)

      Pages: 473-474

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Cutaneous pemphigus vulgaris with skin features similar to the classic mucocutaneous type : a case report and review of the literature2008

    • Author(s)
      Shinkuma S, Ito K
    • Journal Title

      Clinical Experiment of Dermatology 33(6)

      Pages: 724-728

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Presentation] Surviving animal model of dystrophic epidermolysis bullosa with a human mutated gene toward tailored therapies2008

    • Author(s)
      伊藤圭, 澤村大輔, 西江渉, 中村秀樹, 後藤真希, Jouni Uitto, Christpher P Denton, 中島修, 清水宏
    • Organizer
      第30回水疱症研究会
    • Place of Presentation
      東京
    • Year and Date
      2008-10-26
    • Related Report
      2009 Final Research Report
  • [Presentation] Surviving animal model of dystrophic epidermolysis bullosa with a human mutated gene toward tailored therapies2008

    • Author(s)
      Ito K
    • Organizer
      第30回水疱症研究会
    • Place of Presentation
      ホテルサンルートプラザ新宿・東京
    • Year and Date
      2008-10-26
    • Related Report
      2008 Annual Research Report

URL: 

Published: 2008-04-01   Modified: 2016-04-21  

Information User Guide FAQ News Terms of Use Attribution of KAKENHI

Powered by NII kakenhi