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Establishing a non-invasive real-time Ca2+ imaging with optogenetics-induced muscle stimulation in skeletal myotubes using DMD patient-derived iPSC

Research Project

Project/Area Number 18K14663
Research Category

Grant-in-Aid for Early-Career Scientists

Allocation TypeMulti-year Fund
Review Section Basic Section 43040:Biophysics-related
Research InstitutionKyoto University

Principal Investigator

Uchimura Tomoya  京都大学, iPS細胞研究所, 特定研究員 (50815222)

Project Period (FY) 2018-04-01 – 2021-03-31
Project Status Completed (Fiscal Year 2020)
Budget Amount *help
¥4,030,000 (Direct Cost: ¥3,100,000、Indirect Cost: ¥930,000)
Fiscal Year 2020: ¥780,000 (Direct Cost: ¥600,000、Indirect Cost: ¥180,000)
Fiscal Year 2019: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000)
Fiscal Year 2018: ¥2,210,000 (Direct Cost: ¥1,700,000、Indirect Cost: ¥510,000)
KeywordsiPS細胞 / 骨格筋 / 収縮 / 筋疾患 / 光刺激 / 収縮力 / 筋疲労 / スクリーニング / オプトジェネティクス / カルシウム / Skeletal muscle / Optogenetic / Calcium / iPSC / Screening
Outline of Final Research Achievements

Duchenne muscular dystrophy is characterized by the reduction of contractile performance. For the drug development, it is necessary to establish a disease model that can assay muscle functions as well as many samples in the same plate. In this study, we established an in vitro model evaluating contractile performance using optogenetics and DMD-derived iPSC. Compared to electrical field systems, using optogenetics enables us to assay in high-throughput compatible system as well as measuring calcium mobilization. In the future, it is highly expected to identify new candidates of compounds to treat DMD through performing screening using middle-scale library.

Academic Significance and Societal Importance of the Research Achievements

Duchenne muscular dystrophyは依然として、有効な治療薬が限られている筋疾患の1つであり、iPS細胞を用いた新規創薬が期待されている。本研究では、従来使用されてきた電気刺激に代わり光遺伝子技術を用いる事で、患者病態を再現している筋収縮力の低下を指標とした創薬スクリーニングをiPS細胞レベルで可能とした。その結果、より多くの化合物を評価する事が可能となり、新規化合物の発見が期待される。

Report

(4 results)
  • 2020 Annual Research Report   Final Research Report ( PDF )
  • 2019 Research-status Report
  • 2018 Research-status Report
  • Research Products

    (1 results)

All 2020

All Patent(Industrial Property Rights) (1 results)

  • [Patent(Industrial Property Rights)] 光刺激を用いた骨格筋筋細胞の成熟化と収縮運動を指標とし てスクリーニング法の開発2020

    • Inventor(s)
      内村智也、櫻井英俊
    • Industrial Property Rights Holder
      内村智也、櫻井英俊
    • Industrial Property Rights Type
      特許
    • Filing Date
      2020
    • Related Report
      2020 Annual Research Report

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Published: 2018-04-23   Modified: 2022-01-27  

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