Human FVIII expression using a HAC vector toward stem cell-mediated gene therapy for hemophilia A

• Project/Area Number: 20890130
• Research Category: Grant-in-Aid for Young Scientists (Start-up)
• Allocation Type: Single-year Grants
• Research Field: Hematology
• Research Institution: Tottori University
• Principal Investigator: KUROSAKI Hajime Tottori University, 大学院・医学系研究科, 助教 (70464295)
• Project Period (FY): 2008 – 2009
• Project Status: Completed (Fiscal Year 2009)
• Budget Amount: ¥3,302,000 (Direct Cost: ¥2,540,000、Indirect Cost: ¥762,000); Fiscal Year 2009: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000); Fiscal Year 2008: ¥1,742,000 (Direct Cost: ¥1,340,000、Indirect Cost: ¥402,000)
• Keywords: 染色体 / 血友病 / iPS細胞 / ヒト人工染色体ベクター / 細胞補充

Research Abstract

We sought to elucidate the potential usefulness of a human artificial chromosome (HAC) vector carrying the human FVIII cDNA for gene therapy of Hemophilia A. We have demonstrated the production and secretion of FVIII using the HAC vector carrying multi copies of FVIII in the hemophiliac mouse derived stem cell. This develops promising gene therapies for hemophilia A.

Research Products

• [Journal Article] Effects of Tritrichomonas muris on the Mouse Intestine: A Proteomic Analysis. (2009)
  • Author(s): Kashiwagi A, Kurosaki H, Luo H, Yamamoto H, Oshimura M, Shibahara T.
  • Journal Title: Experimental Animals Vol.58,No.5 Pages: 537-542
  • NAID: 130000122265
  • Peer Reviewed
• [Presentation] 血友病A型細胞補充療法を目指したヒト第VIII因子発現HACベクターの有用性の検討 (2010)
  • Author(s): 黒崎創
  • Organizer: 第9回日本再生医療学会総会
  • Place of Presentation: 国際会議場(広島)
  • Year and Date: 2010-03-18
• [Presentation] Human FVIII expression using a HAC vector toward stem cell-mediated gene therapy for hemophilia A (2009)
  • Author(s): Hajime Kurosaki
  • Organizer: American Society of Human Genetics
  • Place of Presentation: Honolulu, Hawaii
  • Year and Date: 2009-10-22
• [Presentation] 血友病A型モデルマウス由来ntES細胞における第VIII因子発現HACベクターの有用性 (2009)
  • Author(s): 宇野愛海
  • Organizer: 第7回幹細胞シンポジウム
  • Place of Presentation: 慶應義塾大学(東京都)
  • Year and Date: 2009-05-15
• [Presentation] 血友病A型モデルマウス由来ntES細胞における第VIII因子発現HACベクターの有用性 (2009)
  • Author(s): 宇野愛海
  • Organizer: 第7回幹細胞シンポジウム
  • Place of Presentation: 應慶義塾大学(東京都)
  • Year and Date: 2009-05-15
• [Presentation] Human FVIII Expression Using a HAC Vector Toward Gene Therapy for Hemophilia A (2008)
  • Author(s): Hajime Kurosaki
  • Organizer: American Society of Gene Therapy 11t h Annual Meeting
  • Place of Presentation: Boston, Massachusetts in USA
  • Year and Date: 2008-05-31
• [Presentation] Human FVIII Expression Using a HAC Vector Toward Gene Therapy for Hemophilia A (2008)
  • Author(s): Hajime Kurosaki
  • Organizer: American Society of Gene Therapy 11^<th> Annua1 Meeting
  • Place of Presentation: Boston, Massachusetts in USA
  • Year and Date: 2008-05-31
• [Book] 幹細胞研究の最近の進歩幹細胞へのヒト人工染色体導入-医学・薬学への応用- (2009)
  • Author(s): 黒崎創
  • Publisher: 最新医学社
• [Book] 幹細胞研究の最近の進歩 幹細胞へのヒト人工染色体導入-医学・薬学への応用- (2009)
  • Author(s): 黒崎 創
  • Publisher: 最新医学社