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

2010 Fiscal Year Final Research Report

• Project/Area Number: 20591003
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Single-year Grants
• Section: 一般
• Research Field: Neurology
• Research Institution: Kumamoto 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
• 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

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.

Research Products

• [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
  • 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
  • Peer Reviewed
• [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.
  • Year and Date: 20100519-20100522
• [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
  • Year and Date: 20080626-20080627
• [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
• [Remarks] ホームページ等
  • URL: http://www.kumamoto-neuro.jp/index.html