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Therapeutical strategy of Spinal muscular atrophy by RNA virus vector delivery to human iPS-derived neural cells

Research Project

Project/Area Number 23659530
Research Category

Grant-in-Aid for Challenging Exploratory Research

Allocation TypeMulti-year Fund
Research Field Pediatrics
Research InstitutionMicrobial Chemistry Research Foundation

Principal Investigator

ARAKAWA Masayuki  公益財団法人微生物化学研究会, 微生物化学研究所, 研究員 (90398868)

Co-Investigator(Renkei-kenkyūsha) SAITO Kayoko  東京女子医科大学, 医学部, 教授 (90138834)
Project Period (FY) 2011 – 2013
Project Status Completed (Fiscal Year 2013)
Budget Amount *help
¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2013: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000)
Fiscal Year 2012: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2011: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Keywords脊髄性筋萎縮症 / ポリオウイルス / 運動神経細胞 / 人工多能性幹細胞 / RNAウイルス / RNAウイルスベクター
Research Abstract

Spinal muscular atrophy (SMA), a genetic neuromuscular disorder, leads to motor neuron degeneration. SMA is caused by reduced production of the survival of motor neuron (SMN) protein resulting from the homozygous deletion or mutation of the SMN1 gene. To date, however, it remains unclear how SMN protein deficiency causes the selective spinal motor neuron death seen in SMA because of a lack of a method to express SMN proteins by the appropriate delivery to the spinal cord. Therefore, an effective treatment of SMA does not presently exist. This study was focused on the potential of a neurotropic RNA virus, poliovirus (PV)-based vector as a novel therapeutic delivery system of exogenous protein in human iPS-derived neural cells and SMA-derived cells.In this study, PV-based expression vector delivery method exhibited an insight into the GFP or GFP-SMN protein expression during an early stage of human iPS-derived neural cells and SMA patient-derived cells.

Report

(4 results)
  • 2013 Annual Research Report   Final Research Report ( PDF )
  • 2012 Research-status Report
  • 2011 Research-status Report
  • Research Products

    (4 results)

All 2013 Other

All Presentation (2 results) Remarks (2 results)

  • [Presentation] 神経変性疾患の治療を目指した外来遺伝子発現ポリオウイルスベクターの開発研究2013

    • Author(s)
      荒川正行、滝沢直己、藤原俊伸、斎藤加代子、野本明男
    • Organizer
      第61回日本ウイルス学会学術集会
    • Place of Presentation
      神戸国際展示場(神戸市)
    • Year and Date
      2013-11-10
    • Related Report
      2013 Final Research Report
  • [Presentation] 神経変性疾患の治療を目指した外来遺伝子発現ポリオウイルスベクターの開発研究

    • Author(s)
      荒川正行、滝沢直己、藤原俊伸、斎藤加代子、野本明男
    • Organizer
      第61回日本ウイルス学会学術集会
    • Place of Presentation
      神戸国際展示場(神戸市)
    • Related Report
      2013 Annual Research Report
  • [Remarks] 公益財団法人微生物化学研究会 微生物化学研究所

    • URL

      http://www.bikaken.or.jp

    • Related Report
      2013 Final Research Report
  • [Remarks] 公益財団法人微生物化学研究会 微生物化学研究所

    • URL

      http://www.bikaken.or.jp/

    • Related Report
      2013 Annual Research Report

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Published: 2011-08-05   Modified: 2019-07-29  

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