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Development of gene and cell therapy for muscular dystrophies.

Research Project

Project/Area Number 24591284
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeMulti-year Fund
Section一般
Research Field Neurology
Research InstitutionNational Center of Neurology and Psychiatry

Principal Investigator

KIMURA En  独立行政法人国立精神・神経医療研究センター, トランスレーショナルメディカルセンター, 室長 (60433025)

Project Period (FY) 2012-04-01 – 2015-03-31
Project Status Completed (Fiscal Year 2014)
Budget Amount *help
¥5,330,000 (Direct Cost: ¥4,100,000、Indirect Cost: ¥1,230,000)
Fiscal Year 2014: ¥130,000 (Direct Cost: ¥100,000、Indirect Cost: ¥30,000)
Fiscal Year 2013: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
Fiscal Year 2012: ¥3,120,000 (Direct Cost: ¥2,400,000、Indirect Cost: ¥720,000)
Keywords筋ジストロフィー / 遺伝子治療 / レンチウイルスベクター / 幹細胞移植 / 幹細胞移植治療 / 再生医療
Outline of Final Research Achievements

Series of Lentiviral vectors were prepared; each carrying different-type of full- and short-version of dystrophin, marker gene, myogenic transcriptional factors, under the control of various combinations of muscle specific promoters and the miR target sequences. The myoD-ER(T), fusion gene with myoD and modified estrogen receptor biding domain for Tamoxifen, was successfully introduced into dermal fibroblasts by lentiviral transduction, and covert themselves to myogenic cells. These cell therapy resources are candidates of genetic modifications by not only viral vectors based technologies and newly developed gene-editing technologies.

Report

(4 results)
  • 2014 Annual Research Report   Final Research Report ( PDF )
  • 2013 Research-status Report
  • 2012 Research-status Report
  • Research Products

    (38 results)

All 2015 2014 2013 2012 Other

All Journal Article (14 results) (of which Peer Reviewed: 14 results,  Open Access: 3 results,  Acknowledgement Compliant: 3 results) Presentation (15 results) (of which Invited: 7 results) Book (6 results) Remarks (3 results)

  • [Journal Article] The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations.2015

    • Author(s)
      1)Bladen CL, Salgado D, Monges S, Foncuberta ME, …, Kimura E, …, Bushby K, Verschuuren J, Aartsma-Rus A, Beroud C, Lochmuller H.
    • Journal Title

      Hum mutat.

      Volume: 36 Issue: 4 Pages: 395-402

    • DOI

      10.1002/humu.22758

    • Related Report
      2014 Annual Research Report
    • Peer Reviewed / Open Access / Acknowledgement Compliant
  • [Journal Article] Nationwide patient registry for GNE myopathy in Japan.2014

    • Author(s)
      Mori-Yoshimura M, Hayashi YK, Yonemoto N, Nakamura H, Murata M, Takeda S, Nishino I, Kimura E.
    • Journal Title

      Orphanet J Rare Dis.

      Volume: 9 Issue: 1 Pages: 150-150

    • DOI

      10.1186/s13023-014-0150-4

    • Related Report
      2014 Annual Research Report
    • Peer Reviewed / Open Access / Acknowledgement Compliant
  • [Journal Article] The infrastructure for the clinical research of muscular dystrophies: Remudy and MDCTN2014

    • Author(s)
      木村 円, 中村治雅, 三橋里美, 竹内芙実, 森まどか, 清水玲子, 小牧宏文, 林由起子, 西野一三, 川井 充, 武田伸一
    • Journal Title

      Rinsho Shinkeigaku

      Volume: 54 Issue: 12 Pages: 1069-1070

    • DOI

      10.5692/clinicalneurol.54.1069

    • NAID

      130004921235

    • ISSN
      0009-918X, 1882-0654
    • Related Report
      2014 Annual Research Report
    • Peer Reviewed / Open Access / Acknowledgement Compliant
  • [Journal Article] Prednisolone improves walking in Japanese Duchenne muscular dystrophy patients2013

    • Author(s)
      Takeuchi F, Yonemoto N, Nakamura H, Shimizu R, Komaki H, Mori-Yoshimura M, Hayashi YK, Nishino I, Kawai M, Kimura E, Takeda S
    • Journal Title

      J Neurol

      Volume: 260 Issue: 12 Pages: 3023-3029

    • DOI

      10.1007/s00415-013-7104-y

    • Related Report
      2013 Research-status Report
    • Peer Reviewed
  • [Journal Article] The TREAT-NMD Duchenne muscular dystrophy registries: conception, design, and utilization by industry and academia.2013

    • Author(s)
      Bladen CL, Rafferty K, ..., Kimura E, ..., Lochmuller H.
    • Journal Title

      Hum mutat.

      Volume: 34(11) Issue: 11 Pages: 1449-1457

    • DOI

      10.1002/humu.22390

    • Related Report
      2013 Research-status Report
    • Peer Reviewed
  • [Journal Article] Characteristics of Japanese Duchenne and Becker muscular dystrophy patients in a novel Japanese national registry of muscular dystrophy (Remudy).2013

    • Author(s)
      Nakamura H, Kimura E†, Mori-Yoshimura M, Komaki H, Matsuda Y, Goto K, Hayashi YK, Nishino I, Takeda SI, Kawai M.
    • Journal Title

      Orphanet J Rare Dis.

      Volume: 8(1) Issue: 1 Pages: 60-60

    • DOI

      10.1186/1750-1172-8-60

    • Related Report
      2013 Research-status Report 2012 Research-status Report
    • Peer Reviewed
  • [Journal Article] Optineurin is potentially associated with TDP-43 and involved in the pathogenesis of inclusion body myositis.2013

    • Author(s)
      Yamashita S., Kimura E., Tawara N., Sakaguchi H., Nakama T., Maeda Y., Hirano T., Uchino M., & Ando Y.
    • Journal Title

      Neuropathol. Appl. Neurobiol.

      Volume: 39 Issue: 4 Pages: 406-416

    • DOI

      10.1111/j.1365-2990.2012.01297.x

    • Related Report
      2013 Research-status Report 2012 Research-status Report
    • Peer Reviewed
  • [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
      2013 Research-status Report 2012 Research-status Report
    • Peer Reviewed
  • [Journal Article] Efficient and reproducible myogenic differentiation from human iPS cells: prospects for modeling Miyoshi Myopathy in vitro.2013

    • Author(s)
      Tanaka, A., et al
    • Journal Title

      PLoS ONE

      Volume: 8(4) Issue: 4 Pages: e61540-e61540

    • DOI

      10.1371/journal.pone.0061540

    • Related Report
      2013 Research-status Report 2012 Research-status Report
    • Peer Reviewed
  • [Journal Article] Heatstroke in patients with Parkinson's disease2012

    • Author(s)
      Satoshi Yamashita
    • Journal Title

      Neurological Sciences

      Volume: 33 Issue: 3 Pages: 685-687

    • DOI

      10.1007/s10072-011-0842-7

    • Related Report
      2012 Research-status 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 Research-status Report
    • Peer Reviewed
  • [Journal Article] Long-term outcome of polymyositis treated with high single-dose alternate-day prednisolone therapy2012

    • Author(s)
      Makoto Uchino
    • Journal Title

      European Neurology

      Volume: 68 Issue: 2 Pages: 117-121

    • DOI

      10.1159/000338474

    • Related Report
      2012 Research-status Report
    • Peer Reviewed
  • [Journal Article] Myasthenic crisis patients who require intensive care unit management2012

    • Author(s)
      Hideya Sakaguchi
    • Journal Title

      Muscle and Nerve

      Volume: 46 Issue: 3 Pages: 440-442

    • DOI

      10.1002/mus.23445

    • Related Report
      2012 Research-status Report
    • Peer Reviewed
  • [Journal Article] The association between dermatomyositis and papillary thyroid cancer: a case report.2012

    • Author(s)
      Ikeda T, Kimura E, Hirano T, Uchino M
    • Journal Title

      Rheumatol Int.

      Volume: 32(4) Issue: 4 Pages: 959-61

    • DOI

      10.1007/s00296-010-1763-0

    • Related Report
      2012 Research-status Report
    • Peer Reviewed
  • [Presentation] Remudy, Japanese national registry for neuromuscular diseases, as a clinical utility model for various rare disease registries with international harmonization2015

    • Author(s)
      Kimura E, Nakamura H, Mitsuhashi S, Mori-Yoshimura M, Takahashi PM, Takeuchi F, Wu S, Zheng Y, Matsumura T, Komaki H, Nishino I, Kawai M, Dawkins H, Béroud C, Lochmüller H, Takeda S, and Remudy Muscular Dystrophy Clinical Research Group
    • Organizer
      14th AOMC Annual Scientific Meeting
    • Place of Presentation
      Shangri-La Hotel, Bangkok, Thailand
    • Year and Date
      2015-03-01 – 2015-03-04
    • Related Report
      2014 Annual Research Report
  • [Presentation] 患者登録システム, Remudy – の意義2014

    • Author(s)
      木村 円
    • Organizer
      第1回筋ジストロフィー医療研究会
    • Place of Presentation
      国立病院機構講堂, 東京
    • Year and Date
      2014-10-24 – 2014-10-25
    • Related Report
      2014 Annual Research Report
    • Invited
  • [Presentation] National registry of Japanese dystrophinopathy patients: Remudy2014

    • Author(s)
      Takeuchi F, Nakamura H, Mitsuhashi S, Mori-Yoshimura M, Hayashi YK, Shimizu R, Komaki H, Nishino I, Kawai M, Takeda S, Kimura E.
    • Organizer
      19th International WMS Congress
    • Place of Presentation
      the Academic Center of Berlin, in the Langenbeck-Virchow-Haus, Berlin, Germany
    • Year and Date
      2014-10-07 – 2014-10-11
    • Related Report
      2014 Annual Research Report
  • [Presentation] DMD/BMD patient registry in Japan: Remudy2014

    • Author(s)
      Kimura E, Nakamura H, Hayashi YK, Mori-Yoshimura M, Takeuchi F, Shimizu R, Komaki H, Nishino I, Kawai M, Takeda S
    • Organizer
      13th International Congress on Neuromuscular Diseases ICNMD
    • Place of Presentation
      Acroplis Convention Centre, Nice, France
    • Year and Date
      2014-07-05 – 2014-07-10
    • Related Report
      2014 Annual Research Report
  • [Presentation] 筋ジストロフィーの臨床開発を推進する研究基盤:RemudyとMDCTN2014

    • Author(s)
      木村 円, 中村治雅, 三橋里美, 竹内芙実, 森まどか, 清水玲子, 小牧宏文, 林由起子, 西野一三, 川井 充, 武田伸一
    • Organizer
      第55回日本神経学会学術大会
    • Place of Presentation
      福岡国際会議場, 福岡
    • Year and Date
      2014-05-21 – 2014-05-24
    • Related Report
      2014 Annual Research Report
    • Invited
  • [Presentation] The CARE-NMD survey in Japan,2013

    • Author(s)
      Kimura E.
    • Organizer
      The CARE-NMD International Conference on DMD
    • Place of Presentation
      Hilton Westend,Budapest, Hangary
    • Related Report
      2013 Research-status Report
    • Invited
  • [Presentation] Neuromuscular disease Registry2013

    • Author(s)
      Kimura E.
    • Organizer
      第一回上海DMDトランスレーショナル医学と健康介護シンポジューム
    • Place of Presentation
      Children's Hosital of Fudan University 上海, 中国
    • Related Report
      2013 Research-status Report
    • Invited
  • [Presentation] Current status of dystrophinopathy patient registry in Japan: Remudy.2013

    • Author(s)
      Kimura E, Nakamura H, Hayashi YK, Mori-Yoshimura M, Takeuchi F, Shimizu R, Komaki H, Nishino I, Kawai M, Takeda S
    • Organizer
      The 18th International Congress of the World Muscle Society
    • Place of Presentation
      Asilomar Conference Grounds,California,USA
    • Related Report
      2013 Research-status Report
  • [Presentation] Current status of patient registration in Japan: REMUDY - Infrastructure for new drug development to treat muscular dystrophy2013

    • Author(s)
      Kimura E, Nakamura H, Hayashi YK, Mori-Yoshimura M, Takeuchi F, Shimizu R, Komaki H, Nishino I, Kawai M, Takeda S
    • Organizer
      The 12th Annual Scientific Meeting of the Asian and Oceanian Myology Center (AOMC)
    • Place of Presentation
      Sofitel Xi'an on Renmin Square Xi’an, China
    • Related Report
      2013 Research-status Report
  • [Presentation] 希少疾患レジストリー:国際協調と臨床開発における役割 ー Remudyの現状と目指すもの2013

    • Author(s)
      木村 円
    • Organizer
      第34回日本臨床薬理学会学術総会
    • Place of Presentation
      東京国際フォーラム(東京)
    • Related Report
      2013 Research-status Report
    • Invited
  • [Presentation] Remudy・DMD/BMD患者情報登録の現状2013

    • Author(s)
      木村 円, 林由起子, 森まどか, 清水玲子, 小牧宏文, 西野一三, 川井 充, 武田伸一
    • Organizer
      第54回日本神経学会学術大会
    • Place of Presentation
      東京国際フォーラム(東京)
    • Related Report
      2013 Research-status Report
  • [Presentation] INFRASTRUCTURE FOR NEW DRUG DEVELOPMENT TO TREAT MUSCULAR DYSTROPHY - CURRENT STATUS OF PATIENT REGISTRATION IN JAPAN: REMUDY.2012

    • Author(s)
      Kimura, E, Nakamura H, Hayashi YK, Mori MY, Komaki H, Nishino I, Kawai M, Takeda S
    • Organizer
      ICORD 2012
    • Place of Presentation
      東京大学 駒場リサーチキャンパス コンベンション・ホール
    • Related Report
      2012 Research-status Report
  • [Presentation] DMD/BMD 登録制度.2012

    • Author(s)
      木村 円
    • Organizer
      2012国际DMD康复与治疗大会暨第一届DMD医患大会
    • Place of Presentation
      北京, 中国General Hospital of Chinese Armed Police Force
    • Related Report
      2012 Research-status Report
    • Invited
  • [Presentation] Exciting developments in the Japanese neuromuscular field2012

    • Author(s)
      Kimura E, Nakamura H, Hayashi YK, Mori-Yoshimura M, Komaki H, Shimizu R, Ogata K, Matsumura T, Takahashi MP, Takada H, Nishino I, Kawai M, Takeda S
    • Organizer
      6th TREAT-NMD Curators’ & 5th TGDOC Meeting
    • Place of Presentation
      Istanbul, Turkey Kalyon Hotel Sahilyolu
    • Related Report
      2012 Research-status Report
    • Invited
  • [Presentation] Infrastructure for new drug development to treat muscular dystrophy-Current status of patient registration in japan:Remudy2012

    • Author(s)
      Kimura E, Nakamura H, Hayashi YK, Mori-Yoshimura M, Shimizu R, Komaki H, Nishino I, Kawai M, Takeda S
    • Organizer
      17th International Congress of the World Muscle Society
    • Place of Presentation
      Perth, Australia The Perth Convention and Exhibition Center
    • Related Report
      2012 Research-status Report
  • [Book] Clinical Neuroscience2015

    • Author(s)
      木村 円
    • Publisher
      中外医学社
    • Related Report
      2014 Annual Research Report
  • [Book] BRAIN and NERVE2014

    • Author(s)
      木村 円, 中村治雅, 西野一三
    • Publisher
      医学書院
    • Related Report
      2014 Annual Research Report
  • [Book] 今日の神経疾患治療指針 第2版2013

    • Author(s)
      木村円, 武田伸一
    • Total Pages
      1100
    • Publisher
      医学書院
    • Related Report
      2013 Research-status Report
  • [Book] 中外医学社2013

    • Author(s)
      木村円
    • Total Pages
      206
    • Publisher
      中外医学社
    • Related Report
      2013 Research-status Report
  • [Book] 今日の神経疾患治療指針 第2版 進行性筋ジストロフィー(ジストロフィノパチー)2013

    • Author(s)
      木村円, 武田伸一
    • Total Pages
      1100
    • Publisher
      医学書院
    • Related Report
      2012 Research-status Report
  • [Book] 筋疾患のみかた,考えかた Remudyによる患者登録2013

    • Author(s)
      木村円
    • Total Pages
      820
    • Publisher
      中外医学社
    • Related Report
      2012 Research-status Report
  • [Remarks] Remudy

    • URL

      http://www.remudy.jp

    • Related Report
      2013 Research-status Report
  • [Remarks] 国立精神・神経医療研究センター トランスレーショナル・メディカルセンター

    • URL

      http://www.ncnp.go.jp/tmc/

    • Related Report
      2013 Research-status Report
  • [Remarks] 神経・筋疾患患者情報登録 Remudy

    • URL

      http://www.remudy.jp

    • Related Report
      2012 Research-status Report

URL: 

Published: 2013-05-31   Modified: 2019-07-29  

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