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Amino acid substitution without genetic modification

Research Project

Project/Area Number 15H05491
Research Category

Grant-in-Aid for Young Scientists (A)

Allocation TypeSingle-year Grants
Research Field Bio-related chemistry
Research InstitutionNagoya University

Principal Investigator

Hagihara Shinya  名古屋大学, 理学研究科, 准教授 (80373348)

Research Collaborator YAMASHITA Shun  
Project Period (FY) 2015-04-01 – 2018-03-31
Project Status Completed (Fiscal Year 2017)
Budget Amount *help
¥24,310,000 (Direct Cost: ¥18,700,000、Indirect Cost: ¥5,610,000)
Fiscal Year 2017: ¥7,670,000 (Direct Cost: ¥5,900,000、Indirect Cost: ¥1,770,000)
Fiscal Year 2016: ¥7,670,000 (Direct Cost: ¥5,900,000、Indirect Cost: ¥1,770,000)
Fiscal Year 2015: ¥8,970,000 (Direct Cost: ¥6,900,000、Indirect Cost: ¥2,070,000)
Keywords革新的遺伝子治療 / 核酸医薬 / アンチセンス / ナンセンス変異 / リードスルー / 核酸 / 糖 / リボソーム結合分子 / 生体分子 / 遺伝情報変換
Outline of Final Research Achievements

A nonsense mutation is a genetic mutation that converts a codon coding an amino acid to the stop codon called premature termination codon (PTC). This mutation causes a variety of diseases including Duchenne muscular dystrophy and cystic fibrosis. One of the potential methods for treating such diseases is readthrough therapy that is accomplished by the incorporation of an amino acid to PTC during translation and hence produces the full-length proteins. So far, aminoglycosides and other synthetic compounds that reduce the translation fidelity have been utilized to readthrough therapy. However, their toxicity due to the non-specific induction of inaccurate translation hampered their clinical use. In this study, we have developed a novel readthrough strategy that enables the target PTC-specific readthrough.

Report

(4 results)
  • 2017 Annual Research Report   Final Research Report ( PDF )
  • 2016 Annual Research Report
  • 2015 Annual Research Report
  • Research Products

    (9 results)

All 2018 2017 2016 2015 Other

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

  • [Int'l Joint Research] University of Munster(Germany)

    • Related Report
      2016 Annual Research Report
  • [Journal Article] Key Structural Elements of Unsymmetrical Cyanine Dyes for Highly Sensitive Fluorescence Turn-on DNA Probes2017

    • Author(s)
      Kakishi Uno, Taeko Sasaki, Nagisa Sugimoto, Hideto Ito, Taishi Nishihara, Shinya Hagihara, Tetsuya Higashiyama, Narie Sasaki, Yoshikatsu Sato and Kenichiro Itami
    • Journal Title

      Chem. Asian J.

      Volume: 12 Issue: 2 Pages: 233-238

    • DOI

      10.1002/asia.201601430

    • Related Report
      2016 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] High-throughput Assay for Quantification of Aminoglycoside&-Ribosome Interaction2016

    • Author(s)
      Shun Yamashita, Dominik Bergmann, Ayato Sato, Mika Nomoto, Yasuomi Tada, Hans-Ulrich Humpf, Kenichiro Itami and Shinya Hagihara
    • Journal Title

      Chem. Lett.

      Volume: 45 Issue: 9 Pages: 1048-1050

    • DOI

      10.1246/cl.160508

    • NAID

      130005549100

    • Related Report
      2016 Annual Research Report
    • Peer Reviewed / Int'l Joint Research
  • [Journal Article] Probing strigolactone receptors in Striga hermonthica with fluorescence.2015

    • Author(s)
      Tsuchiya, Y., Yoshimura, M., Sato, Y., Kuwata, K., Toh, S., Holbrook-Smith, D., Zhang, H., McCourt, P., Itami, K., Kinoshita, T. and Hagihara, S.
    • Journal Title

      Science

      Volume: 349 Issue: 6250 Pages: 864-869

    • DOI

      10.1126/science.aab3831

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed / Int'l Joint Research
  • [Presentation] Sequence-specific Readthrough of Termination Codon by Antisense Oligonucleotides2018

    • Author(s)
      YAMASHITA, Shun; ITAMI, Kenichiro; HAGIHARA, Shinya
    • Organizer
      日本化学会第98春季年会
    • Related Report
      2017 Annual Research Report
  • [Presentation] 新規リボソーム結合性分子の 迅速スクリーニング2017

    • Author(s)
      山下 隼・佐藤 綾人・伊丹 健一郎・萩原 伸也
    • Organizer
      日本化学会第97春季年会
    • Place of Presentation
      慶応大学
    • Year and Date
      2017-03-16
    • Related Report
      2016 Annual Research Report
  • [Presentation] High throughput screening for the novel translation regulator2017

    • Author(s)
      Shun Yamashita, Ayato Sato, Kenichiro Itami, Shinya Hagihara
    • Organizer
      グリーン自然科学国際教育研究プログラム IGER 2016年度年次報告会
    • Place of Presentation
      名古屋大学
    • Related Report
      2016 Annual Research Report
  • [Presentation] 新規リボソーム結合性分子の探索2015

    • Author(s)
      山下隼・萩原伸也・佐藤綾人・野元美佳・多田安臣・伊丹健一郎
    • Organizer
      第9回バイオ関連化学シンポジウム
    • Place of Presentation
      熊本
    • Year and Date
      2015-09-10
    • Related Report
      2015 Annual Research Report
  • [Presentation] 新規翻訳制御分子の探索に向けたスクリーニング法の開発2015

    • Author(s)
      山下隼・佐藤綾人・野元美佳・多田安臣・伊丹健一郎・萩原伸也
    • Organizer
      第3回バイオ関連化学シンポジウム若手フォーラム
    • Place of Presentation
      熊本
    • Year and Date
      2015-09-09
    • Related Report
      2015 Annual Research Report

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Published: 2015-04-16   Modified: 2021-01-27  

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