Drug screening system development for skeletal muscle diseases using human iPSC

2018 Fiscal Year Final Research Report

• Project/Area Number: 16K09988
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Pediatrics
• Research Institution: Kyoto University
• Principal Investigator: Awaya Tomonari 京都大学, 医学研究科, 特定助教 (20589593)
• Research Collaborator: Sakurai Hidetoshi 京都大学, iPS細胞研究所, 准教授 ; Hagiwara Masatoshi 京都大学, 医学研究科, 教授
• Project Period (FY): 2016-04-01 – 2019-03-31
• Keywords: iPS細胞 / 骨格筋分化 / 遺伝性筋疾患 / 創薬研究

Outline of Final Research Achievements

iPSCs can differentiate into various types of somatic cells; however, it is usually difficult to obtain homogenous and stable cells reproducibly. In this research, we compared various methods to maintain undifferentiated iPSCs in regard to their potential to give rise to skeletal muscle cells, aiming to develop suitable culture conditions for high throughput drug screening (HTS). Different culture conditions, especially the use of some extracellular matrices during maintenance culture of undifferentiated cells, enhanced/suppressed skeletal muscle differentiation efficiency. By adjusting culture conditions, we also determined the method to culture skeletal muscle cells in multi-well plate system. It can be used as a HTS platform for skeletal muscle diseases.

Free Research Field

幹細胞生物学

Academic Significance and Societal Importance of the Research Achievements

遺伝性筋疾患は基本的に稀少疾患であり、その病態解析の不十分さや不採算性のため既存の創薬事業には不向きであったが、iPS細胞の登場により研究開発が進められるようになった。一方でiPS細胞研究者の間では培養条件によるばらつきや再現性の困難さが課題となっていた。本研究で行ったiPS細胞の培養条件の最適化は、稀少疾患の創薬研究の推進といった社会的意義の大きいものであり、その過程で得られた細胞－細胞外マトリクスの相互作用に関わる基礎的知見は学術的にも有意義なものである。