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Large gene therapy for dystroglycanopathies

Research Project

Project/Area Number 17H06862
Research Category

Grant-in-Aid for Research Activity Start-up

Allocation TypeSingle-year Grants
Research Field Neurology
Research InstitutionKobe University

Principal Investigator

Otsuka Yoshihisa  神戸大学, 医学部附属病院, 特定助教 (20808860)

Research Collaborator Kanagawa Motoi  
Toda Tatsushi  
Project Period (FY) 2017-08-25 – 2019-03-31
Project Status Completed (Fiscal Year 2018)
Budget Amount *help
¥2,730,000 (Direct Cost: ¥2,100,000、Indirect Cost: ¥630,000)
Fiscal Year 2018: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2017: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Keywords筋ジストロフィー / ジストログリカノパチー / 遺伝子治療 / LARGE
Outline of Final Research Achievements

Regardless of the causative gene, muscular dystrophies characterized by abnormal glycosylation of dystroglycan are collectively referred to as dystroglycanopathy. We studied whether Large gene therapy is commonly effective to dystrocurinopathy regardless of the causative gene in vivo. We performed Large gene therapy to non-severe dystroglycanopathy model mice in which sugar chains remain a certain extent. As a result, improvement of skeletal muscle necrosis was detected in the non-severe type dystroglycanopathy model mice by Large gene therapy. We found that large gene therapy may be applied to non-severe forms of dystroglycanopathy regardless of causative gene.

Academic Significance and Societal Importance of the Research Achievements

いずれの病型においても治療法が確立されていないジストログリカノパチーにおいて,新たな治療法の可能性を見出した.

Report

(3 results)
  • 2018 Annual Research Report   Final Research Report ( PDF )
  • 2017 Annual Research Report

Research Products

(2 results)

All 2018 2017

All Presentation (2 results)

  • [Presentation] LARGE Gene Therapy for α-Dystroglycanopathies2018

    • Author(s)
      Yoshihisa Ohtsuka, Motoi Kanagawa, Tomoko Chiyo, Kazuhiro Kobayashi, Takashi Okada, Shin’ichi Takeda, Tatsushi Toda
    • Organizer
      11th Japanese-French Workshop
    • Related Report
      2018 Annual Research Report
  • [Presentation] ジストログリカノパチーに対するLARGE遺伝子治療2017

    • Author(s)
      大塚喜久
    • Organizer
      日本筋学会第3回学術集会
    • Related Report
      2017 Annual Research Report

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Published: 2017-08-25   Modified: 2020-03-30  

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