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

Helper-dependent adenovirusvector and modified lentiviral vector mediated delivery of dystrophin for gene therapy of muscular dystrophy

Research Project

Project/Area Number 20591003
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeSingle-year Grants
Section一般
Research Field Neurology
Research InstitutionKumamoto University

Principal Investigator

UCHINO Makoto  Kumamoto University, 大学院・生命科学研究部, 教授 (20117336)

Project Period (FY) 2008 – 2010
Project Status Completed (Fiscal Year 2010)
Budget Amount *help
¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2010: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2009: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2008: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Keywords筋ジストロフィー / 遺伝子治療 / ヘルパー依存アデノウイルス / 改変レンチウイルス / full-length dystrophin / dko mouse / whole-body plethysmography / helper-dependent adenovirus vector / lentivirus / lentivirus vector
Research Abstract

We showed that a highly efficient dystrophin-transduction to the mex and dko's diaphragm-achieved by simple intraperitoneal injection of a helper-dependent adenovirus vector (HDAdv) containing the full-length dystrophin expression cassette - provided beneficial results. we carried out the packaging of the full-length dystrophin cDNA in a genetically modified lentivirus with a useful, sufficiently functional titer that was successfully transduced into myogenic cells derived from the transgenic mdx mouse.

Report

(4 results)
  • 2010 Annual Research Report   Final Research Report ( PDF )
  • 2009 Annual Research Report
  • 2008 Annual Research Report
  • Research Products

    (16 results)

All 2011 2010 2009 2008 Other

All Journal Article (7 results) (of which Peer Reviewed: 7 results) Presentation (7 results) Book (1 results) Remarks (1 results)

  • [Journal Article] Muscle fiber type-predominant promoter activity in lentiviral-mediated transgenic mouse.2011

    • Author(s)
      Suga T, Kimura E, Morioka Y, Ikawa M, Li S, Uchino K, Uchida Y, Maeda Y, Chamberlain JS, Uchino M.
    • Journal Title

      PloS One in press

    • NAID

      120006957404

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Rescue from respiratory dysfunction bytransduction of full-length dystrophin to diaphragm via the peritoneal cavity in utrophin/dystrophin double knockout mice.2011

    • Author(s)
      Ishizaki M, Maeda Y, Kawano R, Suga T, Uchida Y, Uchida Y, Uchino K, Yamashita S, Kimura E, Uchino M.
    • Journal Title

      Mol Ther In press

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Muscle fiber type-predominant promoter activity in lentiviral-mediated transgenic mouse2011

    • Author(s)
      Suga T, Kimura E, Morioka Y, Ikawa M, Li S, Uchino K, Uchida Y, Maeda Y, Chamberlain JS, Uchino M.
    • Journal Title

      PloS ONE

      Volume: (In press)

    • NAID

      120006957404

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Rescue from respiratory dysfunction by transduction of full-length dystrophinto diaphragm via the peritoneal cavity in mouse with muscular dystrophy2011

    • Author(s)
      Ishizaki M, Maeda Y, Kawano R, Suga T, Uchida Y, Uchida Y, Uchino K, Yamashita S, Kimura E, Uchino M.
    • Journal Title

      Mol Ther

      Volume: (In press)

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Packaging and delivery of full-length dystrophin in a lentiviral vector2011

    • Author(s)
      Uchino K, kimura E, SugaT, Koide T, Mori A, Uchida Y, Yamashita S, Maeda Y, et al.
    • Journal Title

      PloS ONE

      Volume: (In press)

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Transduction of full-length dystrophin to multiple skeletal muscles improves motor performance and life span in utrophin/dystrophin double knockout mice2008

    • Author(s)
      Kawano R., et al.
    • Journal Title

      Mol Ther 18

      Pages: 825-831

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Mdx respiratory impairment following fibrosis of the diaphragm2008

    • Author(s)
      Ishizaki M., et al.
    • Journal Title

      Neuromuscul disord 18

      Pages: 342-348

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Presentation] Muscle fiber type-predominant promoter activity in lentiviral-mediated transgenic mouse2010

    • Author(s)
      Suga T, Uchino M, et al.
    • Organizer
      18^<th> Annual Congress of the European Society of Gene and Cell Therapy
    • Place of Presentation
      Stella Polare convention center, Milano,イタリア
    • Year and Date
      2010-10-24
    • Related Report
      2010 Annual Research Report
  • [Presentation] Lentivirat vector mediated delivery of full-length dystrophin for gene therapy of muscular dystrophy2010

    • Author(s)
      Kimura E, Uchino M, et al.
    • Organizer
      15^<th> International Congress of the world muscle Society
    • Place of Presentation
      Kumamoto City Hall, Kumamoto,日本
    • Year and Date
      2010-10-15
    • Related Report
      2010 Annual Research Report
  • [Presentation] Lentiviral vector mediated delivery of full-length dystrophin for gene therapy of muscular dystrophy2010

    • Author(s)
      Kimura E, Uchino M, et al.
    • Organizer
      13^<th> Annual Meeting of the American Society of Gene and Cell Therapy
    • Place of Presentation
      Marriott Wardman Park Hotel Washington DC.アメリカ
    • Year and Date
      2010-05-20
    • Related Report
      2010 Annual Research Report
  • [Presentation] Lentiviral vector mediated delivery of full-length dystrophin for gene therapy of muscular dystrophy.2010

    • Author(s)
      Kimura E, Uchino K, Suga T, Ishizaki M, Koide T, Uchida Y, Maeda Y, Yamashita S, Chamberlain JS, Uchino M.
    • Organizer
      13^<th> Annual Meeting of the American Society of Gene and Cell Therapy
    • Place of Presentation
      Marriott Wardman Park Hotel, Washington DC, USA.
    • Related Report
      2010 Final Research Report
  • [Presentation] Transduction of full-length dystrophin to diaphragm ameliorates respiratory dysfunction in severe dystrophic mouse2009

    • Author(s)
      Ishizaki M., et al.
    • Organizer
      The European Society of Gene and Cell Therapy
    • Place of Presentation
      Convention Center at Hannover Faiground, Hannover, Germany
    • Year and Date
      2009-11-23
    • Related Report
      2009 Annual Research Report
  • [Presentation] Gene delivery to dystrophic diaphragm by the helper-dependent adenovirus vector(HDAdv)-mediated full-length dystrophin expression2008

    • Author(s)
      Ishizaki M., et al.
    • Organizer
      American Society of Gene Therapy
    • Place of Presentation
      Boston, USA
    • Year and Date
      2008-05-30
    • Related Report
      2008 Annual Research Report
  • [Presentation] シンポジウム2.新しい神経治療をめぐってーミオパチー領域における最新の治療戦略2008

    • Author(s)
      内野誠
    • Organizer
      第26回日本神経治療学会
    • Place of Presentation
      新横浜プリンスホテル
    • Related Report
      2010 Final Research Report
  • [Book] 最新神経病学(分担:筋肉疾患)2008

    • Author(s)
      内野誠
    • Total Pages
      66
    • Publisher
      金芳堂
    • Related Report
      2010 Final Research Report
  • [Remarks] ホームページ等

    • URL

      http://www.kumamoto-neuro.jp/index.html

    • Related Report
      2010 Final 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