ex vivo gene manipulation of mesenchymal stem cell and application to cell therapy

2010 Fiscal Year Final Research Report

• Project/Area Number: 20200013
• Research Category: Grant-in-Aid for Scientific Research on Innovative Areas (Research a proposed research project)
• Allocation Type: Single-year Grants
• Research Field: Neurology; Neurophysiology and muscle physiology
• Research Institution: Kumamoto University
• Principal Investigator: MAEDA Yasushi 熊本大学, 医学部附属病院, 講師 (60346997)
• Co-Investigator(Kenkyū-buntansha): UCHINO Makoto 熊本大学, 大学院・生命科学研究部, 教授 (20117336) ; KIMURA En 熊本大学, 医学部附属病院, 助教 (60433025)
• Project Period (FY): 2008 – 2010
• Keywords: 筋ジストロフィー / 細胞治療 / 遺伝子操作 / 間葉系幹細胞

Research Abstract

Bone marrow derived mesenchymal stem cells (MSC) could be isolated and cultured from muscular dystrophy model mouse. And it was proved that gene manipulation by helper-dependent adenovirus of was possible without any tricky technique. Moreover, we found MSC’s novel competence to treat muscular dystrophy. MSC has intrinsic ability to modify muscle degeneration and regeneration. We found various growth factors are produced from MSC, and some growth factors of them give effects on muscle regeneration.

Research Products

• [Journal Article] Rescue From Respiratory Dysfunction by transduction of Full-length Dystrophin to Diaphragm via the Peritoneal Cavity in Utrophn/Dystrophin Double Knockout Mice (2011)
  • Author(s): Ishizaki M, Maeda Y, Kawano R, Suga T, Uchida Y, Uchino K, Yamashita S, Kimura E, Uchino M
  • Journal Title: Molecular Therapy Volume: 19(7) Pages: 1230-5
  • Peer Reviewed
• [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, Yamashita S, Maeda Y, Chamberlain JS, Uchino M
  • Journal Title: PLoS One. Volume: 6(3) Pages: e16908
  • Peer Reviewed
• [Presentation] Repetitive allotransplantation of bone marrow stromal cells to muscular dystrophy model mice drastically improves the body build and life span (2011)
  • Author(s): Maeda Y
  • Organizer: 19th Annual Congress of the European Society of Gene and Cell Therapy(ESGCT)
  • Place of Presentation: Brighton, UK
  • Year and Date: 20111027-31
• [Presentation] Muscle fiber type-predominant promoter activity in lentiviral-mediated transgenic mouse (2010)
  • Author(s): Suga T
  • Organizer: 18^<th> Annual Congress of the European Society of Gene and Cell Therapy
  • Place of Presentation: Milano, Italy
  • Year and Date: 20101022-25
• [Presentation] 幹細胞治療による骨格筋再生と筋ジストロフィーの治療研究 (2010)
  • Author(s): 内野 克尚
  • Organizer: 第28 回日本神経治療学会総会
  • Place of Presentation: 横浜(パシフィコ横浜)
  • Year and Date: 20100715-16
• [Presentation] Lentiviral vector mediated delivery of full-length dystrophin for gene therapy of muscular dystrophy (2010)
  • Author(s): 木村 円
  • Organizer: 第16回日本遺伝子治療学会年次学術集会
  • Place of Presentation: 宇都宮(栃木県総合文化センター)
  • Year and Date: 20100701-03
• [Presentation] Lentiviral vector を用いたDuchenne 型筋ジストロフィー遺伝子治療研究 (2010)
  • Author(s): 菅 智宏
  • Organizer: 第51 回日本神経学会総会
  • Place of Presentation: 東京(東京国際フォーラム)
  • Year and Date: 20100520-22
• [Presentation] Transduction of full-length dystrophin to diaphragm ameliorates respiratory dysfunction in severe dystrophic mouse (2010)
  • Author(s): Ishizaki M
  • Organizer: 13^<th> Annual Meeting of the American Society of Gene and Cell Therapy
  • Place of Presentation: Washington DC, USA
  • Year and Date: 20100519-22
• [Presentation] Lentiviral vector mediated delivery of full-length dystrophin for gene therapy of muscular dystrophy (2010)
  • Author(s): Kimura E
  • Organizer: 3^<th> Annual Meeting of the American Society of Gene and Cell Therapy
  • Place of Presentation: Washington DC USA
  • Year and Date: 20100519-22
• [Presentation] Transduction of full-length dystrophin to diaphragm ameliorates respiratory dysfunction in severe dystrophic mouse (2009)
  • Author(s): Ishizaki M
  • Organizer: 17th Annual Congress of the European Society of Gene and Cell Therapy(ESGCT)
  • Place of Presentation: Hannover, Germany, Convention Center at Hannover Fairground
  • Year and Date: 20091120-25
• [Presentation] Full-dystrophin packaging in lentiviral vector for a gene therapy of Duchenne muscular dystrophy (2009)
  • Author(s): Kimura E
  • Organizer: 17th Annual Congress of the European Society of Gene and Cell Therapy(ESGCT)
  • Place of Presentation: Hannover, Germany, Convention Center at Hannover Fairground
  • Year and Date: 20091120-25
• [Presentation] 筋ジストロフィー根本治療に向けての骨髄間質間葉系幹細胞へのウイルスベクターによる遺伝子操作 (2009)
  • Author(s): 前田 寧
  • Organizer: 第27 回日本神経治療学会総会
  • Place of Presentation: 熊本市民会館
  • Year and Date: 20090611-12
• [Presentation] Lentiviral vector を用いたDuchenne 型筋ジストロフィー遺伝子治療研究 (2009)
  • Author(s): 菅 智宏
  • Organizer: 第27 回日本神経治療学会総会
  • Place of Presentation: 熊本市民会館
  • Year and Date: 20090611-12
• [Presentation] 幹細胞治療による骨格筋再生と筋ジストロフィーの治療研究 (2009)
  • Author(s): 木村 円
  • Organizer: 第27回日本神経治療学会総会
  • Place of Presentation: 熊本市民会館
  • Year and Date: 20090611-12
• [Presentation] 横隔膜へのジストロフィン導入による筋ジストロフィーモデルマウスの呼吸機能改善効果 (2009)
  • Author(s): 前田 寧
  • Organizer: 第50 回日本神経学会総会
  • Place of Presentation: 仙台国際センター
  • Year and Date: 20090520-22
• [Presentation] 骨格筋幹細胞をターゲットとしたDuchenne muscular dystrophy の治療研究 (2009)
  • Author(s): 木村 円
  • Organizer: 第50 回日本神経学会総会
  • Place of Presentation: 仙台国際センター
  • Year and Date: 20090520-22