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Gene therapy for neuromuscular disease

Research Project

Project/Area Number 23890163
Research Category

Grant-in-Aid for Research Activity Start-up

Allocation TypeSingle-year Grants
Research Field Neurology
Research InstitutionKumamoto University

Principal Investigator

MORI Akira  熊本大学, 医学部附属病院, 医員 (70608877)

Project Period (FY) 2011 – 2012
Project Status Completed (Fiscal Year 2012)
Budget Amount *help
¥2,210,000 (Direct Cost: ¥1,700,000、Indirect Cost: ¥510,000)
Fiscal Year 2012: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000)
Fiscal Year 2011: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Keywords筋萎縮性側索硬化症 / 小胞体ストレス / Derlin-1 / 変異 SOD1 / 神経細胞死 / アデノ随伴ウイルス / induced Pluripotent Stem (iPS) 細胞 / iPS細胞 / 運動ニューロン / 小胞体関連分野 / 変異SOD1 / 遺伝子治療 / NB2a細胞
Research Abstract

We are intended to develop the gene therapy for neuromusculardisease, especially about amyotrophic lateralsclerosis. At the beginning, We consideredto use NB2a cells, origined from neuroblastoma, previously we have used, but we succeededto establish induced Pluripotent Stem (iPS) cells derived from amyotrophiclateralsclerosis patient skins, and these are considered to reflect pathology much morethan NB2a cells. Therefore, we started using these cells, and were continuing experimentsto prove the participation of endoplasmic reticulum stress. However, maintenancecultivation of an iPS cells is difficult from some technical problems, so we are seekingand solving these problems. If we resolve these problems, we expect that many resultsare obtained.

Report

(3 results)
  • 2012 Annual Research Report   Final Research Report ( PDF )
  • 2011 Annual Research Report
  • Research Products

    (12 results)

All 2013 2012 2011

All Journal Article (8 results) (of which Peer Reviewed: 8 results) Presentation (4 results)

  • [Journal Article] Muscle biopsy findings predictive of malignancy in rare infiltrative dermatomyositis2013

    • Author(s)
      Uchino M, Yamashita S, Uchino K, Mori A, Hara A, Suga T, Hirahara T, Koide T, Kimura E, Yamashita T, Ueda A, Kurisaki R, Suzuki J, Honda S, Maeda Y, Hirano T, Ando Y
    • Journal Title

      Clin Neurol Neurosurg

      Volume: 115(5) Issue: 5 Pages: 603-6

    • DOI

      10.1016/j.clineuro.2012.07.019

    • Related Report
      2012 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Muscle biopsy findings predictive ofmalignancy in rare infiltrativedermatomyositis.2012

    • Author(s)
      Makoto Uchino, Satoshi Yamashita, Katshhisa Uchino, Akira Mori, Akio Hara, Tomohiro Suga, Tomoo Hirahara, Tatsuya Koide, En Kimura, Taro Yamashita, Akihiko Ueda, Reiichi Kurisaki, Junko SuzukiShoji Honda, Yasushi Maeda, Teruyuki Hirano, Yukio Ando. Clin Neurol Neurosurg
    • Journal Title

      Clin Neurol Neurosurg

      Volume: 115 Pages: 603-606

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Significant CMAP decrement byrepetitive nerve stimulation is morefrequent in median than ulnar nerves ofpatients with amyotrophic lateralsclerosis2012

    • Author(s)
      Satoshi Yamashita, HideyaSakaguchi, Akira Mori, En Kimura, MaedaYasushi, Hirano Teruyuki, Uchino Makoto
    • Journal Title

      Muscle Nerve

      Volume: 45(3) Pages: 426-8

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Significant CMAP decrement by repetitive nerve stimulation is more frequent in median than ulnar nerves of patients with amyotrophic lateral sclerosis2012

    • Author(s)
      Satoshi Yamashita, Hideya Sakaguchi, Akira Mori
    • Journal Title

      Muscle Nerve

      Volume: 45 Issue: 3 Pages: 426-428

    • DOI

      10.1002/mus.22301

    • NAID

      120005123115

    • Related Report
      2012 Annual Research Report 2011 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Sporadic juvenile amyotrophic lateralsclerosis caused by mutant FUS/TLS:possible association of mentalretardation with this mutation.2011

    • Author(s)
      SatoshiYamashita, Akira Mori, Hideya Sakaguchi,Tomohiro Suga, Daijiro Ishihara, AkihikoUeda, Taro Yamashita, Yasushi Maeda,Makoto Uchino, Teruyuki Hirano
    • Journal Title

      Journal ofNeurology

      Volume: 259(6) Pages: 1039-44

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Derlin-1 overexpression amelioratesmutant SOD1-induced endoplasmic reticulumstress by reducing mutant SOD1accumulation.2011

    • Author(s)
      Akira Mori, SatoshiYamashita, Katsuhisa Uchino, TomohiroSuga, Tokunori Ikeda, Koutaro Takamatsu,Masatoshi Ishizaki, Tatsuya Koide, EnKimura, Shuji Mita, Yasushi Maeda,Teruyuki Hirano and Makoto Uchino.
    • Journal Title

      Neurochemistry International

      Volume: 58(3) Pages: 344-53

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Sporadic juvenile amyotrophic lateral sclerosis caused by mutant FUS/TLS : possible association of mental retardation with this mutation2011

    • Author(s)
      Satoshi Yamashita, Akira Mori, Hideya Sakaguchi
    • Journal Title

      Journal of Neurplogy

      Volume: 259 Issue: 6 Pages: 1039-1044

    • DOI

      10.1007/s00415-011-6292-6

    • Related Report
      2012 Annual Research Report 2011 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Derlin-1 overexpression ameliorates mutant SOD1-induced endopla smic reticulum stress by reducing mutant SOD1 accumulation2011

    • Author(s)
      Akira Mori, Satoshi Yamashita
    • Journal Title

      Neurochemistry International

      Volume: 58 Issue: 3 Pages: 344-353

    • DOI

      10.1016/j.neuint.2010.12.010

    • Related Report
      2011 Annual Research Report
    • Peer Reviewed
  • [Presentation] Significant CMAP decrement by repetitivenerve stimulation is more frequent inmedian than ulnar nerves of patients withamyotrophic lateral sclerosis2012

    • Author(s)
      Mori A, Yamashita S, Sakaguchi H, KimuraE, Maeda Y, Hirano T, Uchino M, Ando Y
    • Organizer
      23th international Symposium on ALS/MND
    • Place of Presentation
      Chicago, IL, USA
    • Related Report
      2012 Final Research Report
  • [Presentation] Significant CMAP decrement by repetitive nerve stimulation is more frequent in median than ulnar nerves of patients with amyotrophic lateral sclerosis.2012

    • Author(s)
      1. Mori A, Yamashita S, Sakaguchi H, Kimura E, Maeda Y, Hirano T, Uchino M, Ando Y.
    • Organizer
      23th international Symposium on ALS/MND,
    • Place of Presentation
      Chicago, IL, USA
    • Related Report
      2012 Annual Research Report
  • [Presentation] 精神発達遅滞が先行し、FUS/TLS遺伝子のdeletion/frameshift変異を有する孤発性若年性ALSの1例2011

    • Author(s)
      森麗
    • Organizer
      第189回日本神経学会九州地方会
    • Place of Presentation
      久留米大学(福岡)
    • Year and Date
      2011-06-18
    • Related Report
      2011 Annual Research Report
  • [Presentation] Derlin-1過剰発現は変異SOD1蓄積軽減により小胞体ストレスを軽減する2011

    • Author(s)
      森麗
    • Organizer
      第52回日本神経学会学術大会
    • Place of Presentation
      名古屋国際会議場(愛知)
    • Year and Date
      2011-05-19
    • Related Report
      2011 Annual Research Report

URL: 

Published: 2011-09-05   Modified: 2019-07-29  

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