Establishment of the assay system of drug screening for fibrodysplasia ossificans progressiva

• Project/Area Number: 23791636
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Orthopaedic surgery
• Research Institution: Kyoto University
• Principal Investigator: IKEYA Makoto 京都大学, iPS細胞研究所, 准教授 (20442923)
• Project Period (FY): 2011 – 2012
• Project Status: Completed (Fiscal Year 2012)
• Budget Amount: ¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000); Fiscal Year 2012: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000); Fiscal Year 2011: ¥2,210,000 (Direct Cost: ¥1,700,000、Indirect Cost: ¥510,000)
• Keywords: 進行性骨化症線維異形成症 / 進行性骨化性線維異形成症 / 異所性骨化 / BMP受容体

Research Abstract

I have generated iPSCs from patients of fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder characterized by progressive ossification in soft tissues. I have revealed that FOP iPSCs showed increased mineralization and enhanced chondrogenesis in vitro. I also established the assay system of drug screening to suppress the enhanced mineralization of FOP-iPSCs.

Research Products

• [Journal Article] Genetically Matched Human iPS Cells Reveal that Propensity for Cartilage and Bone Differentiation Differs with Clones, not Cell Type of Origin (2013)
  • Author(s): Nasu A
  • Journal Title: PLoS One Volume: 8 Issue: 1 Pages: e53771-e53771
  • DOI: 10.1371/journal.pone.0053771
  • Peer Reviewed
• [Presentation] 人工多能性幹細胞を用いた希少難治性骨疾患への取り組み. (2013)
  • Author(s): 松本佳久、、Edward Hisao、那須輝、浅香勲、大塚隆信、戸口田淳也:
  • Organizer: 第12回日本再生医療学会、一般演題(ポスター)
  • Place of Presentation: 横浜
  • Year and Date: 2013-03-22
• [Presentation] iPS 細胞を用いた難治性骨疾患への取り組み. (2012)
  • Author(s): 松本佳久、池谷真、那須輝、Edward Hisao、大塚隆信、戸口田淳也:
  • Organizer: 第27回日本整形外科学会基礎学術集会、一般口演
  • Place of Presentation: 名古屋
  • Year and Date: 2012-10-26
• [Presentation] Modeling Inherited Skeletal Disease in Human iPS Cells. (2012)
  • Author(s): Schlieve R, Matsumoto Y, Hayashi Y, Kim H, Nguyen T, Sami S, Yamanaka S, Ikeya M,Toguchida J, Conklin B, Hsiao E:
  • Organizer: 第10回国際幹細胞生物学会、ポスター発表、
  • Place of Presentation: 横浜
  • Year and Date: 2012-06-15
• [Presentation] iPS 細胞を用いた難治性骨疾患への取り組み. (2012)
  • Author(s): 松本佳久、池谷真、Edward Hisao、那須輝、浅香勲、大塚隆信、戸口田淳也:
  • Organizer: 第11回日本再生医療学会、一般演題(ポスター)
  • Place of Presentation: 横浜
  • Year and Date: 2012-06-13
• [Presentation] Application of iPS cells to the research of Fibrodysplasia Ossificans Progressiva. (2012)
  • Author(s): Matsumoto Y, Ikeya M, Hsiao E, Schlieve C, Nasu A, Asaka I, Otsuka T, Conklin B,Toguchida J:
  • Organizer: 第10回国際幹細胞生物学会、ポスター発表、
  • Place of Presentation: 横浜、
  • Year and Date: 2012-06-05
• [Presentation] Matsumoto Y, Ikeya M, Nasu A, Asaka I, Otsuka T, Hsiao E, Toguchida J:of fibrodysplasia ossificance progressive. (2011)
  • Author(s): Matsumoto Y, Ikeya M, Nasu A, Asaka I, Otsuka T, Hsiao E, Toguchida J:
  • Organizer: アメリカ骨代謝学会、ポスター発表
  • Place of Presentation: サンディエゴ、アメリカ、
  • Year and Date: 2011-09-16
• [Presentation] Application of iPS cells to the research of fibrodysplasia ossificance progressive (2011)
  • Author(s): Yoshihisa Matsumoto, et al.
  • Organizer: The American Society for Bone and Mineral Research
  • Place of Presentation: San Diego Convention Center（U.S.A.)
• [Presentation] 人工多能性幹細胞を用いた希少難治性骨疾患への取り組み
  • Author(s): 松本佳久
  • Organizer: 第12回 再生医療学会
  • Place of Presentation: パシフィコ横浜（横浜市）
• [Presentation] 人工多能性幹細胞を用いた希少難治性骨疾患への取り組み
  • Author(s): 松本佳久
  • Organizer: 第27 回 日本整形外科学会基礎学術集会
  • Place of Presentation: 名古屋国際会議場（名古屋市）
• [Presentation] Application of iPS cells to the research of Fibrodysplasia Ossificans Progressiva
  • Author(s): Yoshihisa Matsumoto
  • Organizer: ISSCR 10th Annual Meeting
  • Place of Presentation: パシフィコ横浜（横浜市）
• [Presentation] Genetically Matched Human iPS Cells Revealed That the Propensity for iPS Cells to Differentiate into Cartilage Lineage Cells Differs with Clones, but not Cell Type of Origin
  • Author(s): Akira Nasu
  • Organizer: ISSCR 10th Annual Meeting
  • Place of Presentation: パシフィコ横浜（横浜市）
• [Book] iPS細胞-その基礎と臨床応用に向けた展望-、整形・災害外科 (2013)
  • Author(s): 池谷真、松本佳久、横山宏司、戸口田淳也、
  • Publisher: 金原出版株式会社、
• [Book] 整形・災害外科 (2013)
  • Author(s): 池谷 真
  • Total Pages: 10
  • Publisher: 金原出版
• [Book] 遺伝子医学 (2012)
  • Author(s): 戸口田淳也、池谷 真(編集)、メディカルドゥ、
  • Total Pages: 276
  • Publisher: 最新疾患モデルと病態解明・創薬応用研究・細胞医薬創製研究の最前線、
• [Book] 遺伝子医学MOOK (2012)
  • Author(s): 松本佳久、池谷 真、戸口田淳也、メディカルドゥ、
  • Publisher: 難治性骨軟骨疾患罹患者由来iPS細胞を用いた病態再現と治療薬開発の試み、
• [Book] 遺伝子医学MOOK 22号 最新疾患モデルと創薬応用研究、細胞医薬創製研究の最前線 最新疾患モデル動物，ヒト化マウス，モデル細胞，iPS細胞を利用した病態解明から創薬まで (2012)
  • Author(s): 松本 佳久
  • Total Pages: 5
  • Publisher: 株式会社メディカル ドゥ
• [Remarks]
  • URL: http://www.cira.kyoto-u.ac.jp/j/research/ikeya_summary.html
• [Remarks] 京都大学iPS細胞研究所
  • URL: http://www.cira.kyoto-u.ac.jp/j/research/ikeya_summary.html