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Development and application of human neuromuscular junctions

Research Project

Project/Area Number 16H05352
Research Category

Grant-in-Aid for Scientific Research (B)

Allocation TypeSingle-year Grants
Section一般
Research Field Pediatrics
Research InstitutionKyoto University

Principal Investigator

Saito Megumu  京都大学, iPS細胞研究所, 准教授 (90535486)

Co-Investigator(Kenkyū-buntansha) 吉田 路子  京都府立医科大学, 医学(系)研究科(研究院), 助教 (70754571)
Project Period (FY) 2016-04-01 – 2020-03-31
Project Status Completed (Fiscal Year 2019)
Budget Amount *help
¥16,900,000 (Direct Cost: ¥13,000,000、Indirect Cost: ¥3,900,000)
Fiscal Year 2019: ¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000)
Fiscal Year 2018: ¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2017: ¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2016: ¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Keywords多能性幹細胞 / 神経筋接合部 / ヒト多能性幹細胞 / 細胞・組織 / 発生・分化 / 神経科学
Outline of Final Research Achievements

In this study, we constructed a pluripotent-cell based system that simultaneously induces neuromuscular junctions (NMJs) which contain motor neurons, skeletal muscle, and Schwann cells (Lin CY, JCI insight 2019, patent pending). We also showed that spinal muscular atrophy can be used as a prototype for model construction and analysis of NMJ lesions in this system. In the future, we plan to use this unique system to analyze congenital myasthenic syndrome for which no specific treatment exists and spinal muscular atrophy for which the functional analysis of the disease-causing molecule has not been sufficiently advanced.

Academic Significance and Societal Importance of the Research Achievements

今回開発した分化系は機能的なNMJを誘導することができ、形態的あるいは細胞生物学的にNMJの発達を模倣することができることから、疾患モデリングに非常に有用であると考えられる。近年、神経筋疾患の多くが神経筋接合部の異常を伴うことが見いだされつつある。今回の成果は、従来は不可能であったヒトNMJ疾患の病態解析をin vitroでヒトの細胞を用い研究を可能にし、これらの疾患の病態解明や治療法開発に貢献することができる。

Report

(5 results)
  • 2019 Annual Research Report   Final Research Report ( PDF )
  • 2018 Annual Research Report
  • 2017 Annual Research Report
  • 2016 Annual Research Report
  • Research Products

    (19 results)

All 2019 2018 2017 Other

All Int'l Joint Research (1 results) Journal Article (8 results) (of which Int'l Joint Research: 3 results,  Peer Reviewed: 8 results,  Open Access: 3 results) Presentation (8 results) (of which Int'l Joint Research: 2 results,  Invited: 5 results) Patent(Industrial Property Rights) (2 results)

  • [Int'l Joint Research] Academia Sinica, 台湾(その他の国・地域)

    • Related Report
      2019 Annual Research Report
  • [Journal Article] iPSC-derived functional human neuromuscular junctions model the pathophysiology of neuromuscular diseases2019

    • Author(s)
      Lin Chuang-Yu、Yoshida Michiko、Li Li-Tzu、Ikenaka Akihiro、Oshima Shiori、Nakagawa Kazuhiro、Sakurai Hidetoshi、Matsui Eriko、Nakahata Tatsutoshi、Saito Megumu K.
    • Journal Title

      JCI Insight

      Volume: 4 Issue: 18

    • DOI

      10.1172/jci.insight.124299

    • Related Report
      2019 Annual Research Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Generation of a human induced pluripotent stem cell line, BRCi001-A, derived from a patient with mucopolysaccharidosis type I2019

    • Author(s)
      Suga Mika、Kondo Takayuki、Imamura Keiko、Shibukawa Ran、Okanishi Yasue、Sagara Yukako、Tsukita Kayoko、Enami Takako、Furujo Mahoko、Saijo Kaoru、Nakamura Yukio、Osawa Mitsujiro、Saito Megumu K.、Yamanaka Shinya、Inoue Haruhisa
    • Journal Title

      Stem Cell Research

      Volume: 36 Pages: 101406-101406

    • DOI

      10.1016/j.scr.2019.101406

    • Related Report
      2019 Annual Research Report 2018 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Verification and rectification of cell type-specific splicing of a Seckel syndrome-associated ATR mutation using iPS cell model2019

    • Author(s)
      Ichisima Jose、Suzuki Naoya M.、Samata Bumpei、Awaya Tomonari、Takahashi Jun、Hagiwara Masatoshi、Nakahata Tatsutoshi、Saito Megumu K.
    • Journal Title

      Journal of Human Genetics

      Volume: 64 Issue: 5 Pages: 445-458

    • DOI

      10.1038/s10038-019-0574-8

    • Related Report
      2019 Annual Research Report 2018 Annual Research Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Application of induced pluripotent stem cells to primary immunodeficiency diseases2019

    • Author(s)
      Karagiannis Peter、Yamanaka Shinya、Saito Megumu K.
    • Journal Title

      Experimental Hematology

      Volume: 71 Pages: 43-50

    • DOI

      10.1016/j.exphem.2019.01.005

    • Related Report
      2018 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Induced Pluripotent Stem Cells and Their Use in Human Models of Disease and Development.2019

    • Author(s)
      Karagiannis P, Takahashi K, Saito M, Yoshida Y, Okita K, Watanabe A, Inoue H, Yamashita JK, Todani M, Nakagawa M, Osawa M, Yashiro Y, Yamanaka S, Osafune K.
    • Journal Title

      Physiol Rev

      Volume: 99 Issue: 1 Pages: 78-114

    • DOI

      10.1152/physrev.00039.2017

    • Related Report
      2018 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Disease modeling of immunological disorders using induced pluripotent stem cells2018

    • Author(s)
      Saito Megumu K.
    • Journal Title

      Immunological Medicine

      Volume: 41 Issue: 2 Pages: 68-74

    • DOI

      10.1080/13497413.2018.1481583

    • Related Report
      2018 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Rescue of recurrent deep intronic mutation underlying cell type-dependent quantitative NEMO deficiency2018

    • Author(s)
      Boisson Bertrand、Honda Yoshitaka、Ajiro Masahiko、Bustamante Jacinta、Bendavid Matthieu、Gennery Andrew R.、Kawasaki Yuri、Ichishima Jose、Osawa Mitsujiro、Nihira Hiroshi、Shiba Takeshi、Tanaka Takayuki、Chrabieh Maya、Bigio Benedetta、Hur Hong、Itan Yuval、Liang Yupu、Okada Satoshi、et al.
    • Journal Title

      Journal of Clinical Investigation

      Volume: 129 Issue: 2 Pages: 583-597

    • DOI

      10.1172/jci124011

    • Related Report
      2018 Annual Research Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Efficient derivation of sympathetic neurons from human pluripotent stem cells with a defined condition2018

    • Author(s)
      Kirino Kosuke、Nakahata Tatsutoshi、Taguchi Tomoaki、Saito Megumu K.
    • Journal Title

      Scientific Reports

      Volume: 8 Issue: 1 Pages: 12865-12865

    • DOI

      10.1038/s41598-018-31256-1

    • Related Report
      2018 Annual Research Report
    • Peer Reviewed
  • [Presentation] PRULIPOTENT STEM CELL DERIVED MYELOID CELL LINES FOR DISSECTING THE MECHANISM OF AUTOINFLAMMATION.2019

    • Author(s)
      Megumu K. Saito
    • Organizer
      ISSAID2019
    • Related Report
      2019 Annual Research Report
    • Int'l Joint Research
  • [Presentation] Disease Modeling of Pediatric Diseases with iPSCs2019

    • Author(s)
      Megumu K. Saito
    • Organizer
      The 4th TMU International Symposium for Cell Therapy and Regenerative Medicine
    • Related Report
      2019 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] 疾患iPS細胞の病態解析と創薬への応用について2019

    • Author(s)
      齋藤潤
    • Organizer
      再生医療JAPAN
    • Related Report
      2019 Annual Research Report
  • [Presentation] iPS細胞を用いた小児疾患の解析について2019

    • Author(s)
      齋藤潤
    • Organizer
      産学情報交流会
    • Related Report
      2019 Annual Research Report
  • [Presentation] iPS 細胞を用いた病態解析と創薬2018

    • Author(s)
      齋藤潤
    • Organizer
      第45回日本臨床免疫学会総会
    • Related Report
      2017 Annual Research Report
    • Invited
  • [Presentation] iPS細胞を使って 病気の仕組みを知る2018

    • Author(s)
      齋藤潤
    • Organizer
      摂南大学薬学部公開講座
    • Related Report
      2017 Annual Research Report
    • Invited
  • [Presentation] iPS 細胞の医学応用へ向けた研究の現状について2017

    • Author(s)
      齋藤潤
    • Organizer
      京都私立病院協会講演会
    • Place of Presentation
      京都
    • Year and Date
      2017-01-24
    • Related Report
      2016 Annual Research Report
    • Invited
  • [Presentation] 疾患特異的iPS細胞樹立のための 基盤形成事業について2017

    • Author(s)
      齋藤潤
    • Organizer
      東京女子医科大学公開シンポジウム「自閉症・発達障害の成因解明と将来の治療に向けて」
    • Place of Presentation
      東京
    • Year and Date
      2017-01-07
    • Related Report
      2016 Annual Research Report
    • Invited
  • [Patent(Industrial Property Rights)] 多能性幹細胞から人工神経筋接合部を得る方法2017

    • Inventor(s)
      齋藤潤/吉田路子/林壮宇
    • Industrial Property Rights Holder
      齋藤潤/吉田路子/林壮宇
    • Industrial Property Rights Type
      特許
    • Industrial Property Number
      2017-201364
    • Filing Date
      2017
    • Related Report
      2017 Annual Research Report
  • [Patent(Industrial Property Rights)] 神経堤細胞および交感神経細胞の製造方法2017

    • Inventor(s)
      中畑龍俊/斎藤潤/桐野浩輔
    • Industrial Property Rights Holder
      中畑龍俊/斎藤潤/桐野浩輔
    • Industrial Property Rights Type
      特許
    • Industrial Property Number
      2017-088926
    • Filing Date
      2017
    • Related Report
      2017 Annual Research Report

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Published: 2016-04-21   Modified: 2021-02-19  

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