Correction model of hemophilia A mice with multicopy of reprogramming factors and factor VIII gene using HAC

• Project/Area Number: 24790974
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Hematology
• Research Institution: Tottori University
• Principal Investigator: KUROSAKI Hajime 鳥取大学, 医学(系)研究科(研究院), 助教 (70464295)
• Project Period (FY): 2012-04-01 – 2014-03-31
• Project Status: Completed (Fiscal Year 2013)
• Budget Amount: ¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000); Fiscal Year 2013: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000); Fiscal Year 2012: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
• Keywords: 血友病 / 幹細胞 / 再生医療 / 遺伝子 / 再生医学 / 移植・再生医療

Research Abstract

Human artificial chromosome (HAC) vectors show considerable promise for gene therapy applications as they have the capacity to deliver an extremely large genetic region and are stably maintained independent of the host chromosome, thus diminishing risks of insertional mutagenesis. In this study, we have developed HAC vectors for gene therapy of hemophilia.

Research Products

• [Journal Article] An induced pluripotent stem cell-mediated and integration-free factor VIII expression system (2013)
  • Author(s): Yakura Y, Ishihara C, Kurosaki H, Kazuki Y, Komatsu N, Okada Y, Doi T, Takeya H, Oshimura M
  • Journal Title: Biochem Biophys Res Commun Volume: 431(2) Pages: 336-41
  • Peer Reviewed
• [Journal Article] An induced pluripotent stem cell-mediated and integration-free factor VIII expression system. (2013)
  • Author(s): Yakura y, et. al.
  • Journal Title: Biochemical Biophysical Research Communications Volume: 431 Issue: 2 Pages: 336-341
  • DOI: 10.1016/j.bbrc.2012.12.096
  • Peer Reviewed
• [Presentation] ENGINEERED INTEGRATION-FREE iPS CELLS USING ARTIFICIAL CHROMOSOME VECTORS FOR HEMOPHILIA A THERAPY (2014)
  • Author(s): Hajime Kurosaki, Kana Ueda, Teruhiko Suzuki, Yuwna Yakura, Sayaka Fukuhara, Masaharu Hiratsuka, Shoko Takehara, Toko Yoshino, Yasuhiro Kazuki, Takafumi Nakamura and Mitsuo Oshimura
  • Organizer: The 20th annual meeting JSGT 2014
  • Place of Presentation: The Jikei University School of Medicine, Tokyo, Japan
• [Presentation] Toward safe and effective gene- and cell-therapies using human artificial chromosomes and stem cells (2013)
  • Author(s): Narumi Uno, Kana Ueda, Yuichi Iida, Yuna Yakura, Hajime Kurosaki, Katsuhiro Uno, Mitsuhiko Osaki, Tetsuya Ohbayashi, Yasuhiro Kazuki, Masaharu Hiratuka and Mitsuo Oshimura
  • Organizer: International Conference on Genetic Engineering & Genetically Modified Organisms
  • Place of Presentation: DoubleTree by Hilton, Raleigh, NC, USA
  • Year and Date: 2013-08-13
• [Presentation] Systemic cancer virotherapy with MDVV, a combined miRNA-regulated and thymidine kinase-deleted oncolytic vaccinia virus (2013)
  • Author(s): Nitta N., Okada N., Goto I., Yamane M., Nakatake M., Kurosaki H., Nakamura T
  • Organizer: The 19th annual meeting JSGT2013
  • Place of Presentation: CONVENTION CENTER
  • Year and Date: 2013-07-04
• [Presentation] Systemic cancer virotherapy with MDVV, a combined miRNA-regulated and thymidine kinase-deleted oncolytic vaccinia virus (2013)
  • Author(s): Nitta N., Okada N., Goto I., Yamane M., Nakatake M., Kurosaki H., Nakamura T
  • Organizer: The 19th annual meeting JSGT 2013
  • Place of Presentation: OKAYAMA CONVENTION CENTER, Okayama, Japan
• [Presentation] Toward safe and effective gene- and cell-therapies using human artificial chromosomes and stem cells. (2013)
  • Author(s): Narumi Uno, Kana Ueda, Yuichi Iida, Yuna Yakura, Hajime Kurosaki, Katsuhiro Uno,Mitsuhiko Osaki, Tetsuya Ohbayashi, Yasuhiro Kazuki, Masaharu Hiratuka, Mitsuo Oshimura.
  • Organizer: CiRA International Symposium 2013 Raising the Next Generation of Stem Cell Research
  • Place of Presentation: Kyoto University Clock Tower Hall, Japan
• [Presentation] 人工染色体ベクターを用いた新規血友病A 遺伝子治療法の開発 (2012)
  • Author(s): 矢倉裕奈, 黒崎創, 石原千恵, 香月康宏, 土井健史, 松下正, 武谷浩之, 押村光雄
  • Organizer: 第11回日本再生医療学会総会
  • Place of Presentation: 横浜
  • Year and Date: 2012-06-14
• [Presentation] ヒト人工染色体を用いた血小板特異的FVIII 発現iPS細胞の作製-新規治療法確立へ向けて (2012)
  • Author(s): 矢倉裕奈, 黒崎創, 石原千恵, 香月康宏, 土井健史, 武谷浩之, 押村光雄
  • Organizer: 第34回日本血栓止血学会学術集会
  • Place of Presentation: 東京
  • Year and Date: 2012-06-09
• [Presentation] Human artificial chromosomes for gene delivery (2012)
  • Author(s): Oshimura M , Kazuki Y , Hiratsuka M , Ohbayashi T, Iida Y, Uno N, Ueno E, Ueda K, Ohira T, Kurosaki H, Abe S, Kugoh H
  • Organizer: Pep Talk
  • Place of Presentation: USA
  • Year and Date: 2012-01-09
• [Remarks]
  • URL: http://www.med.tottori-u.ac.jp/integbio/523/1165.html