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Regulation of CRISPR-Cas3 deletion length by protein obstacles

Research Project

Project/Area Number 21K15361
Research Category

Grant-in-Aid for Early-Career Scientists

Allocation TypeMulti-year Fund
Review Section Basic Section 48040:Medical biochemistry-related
Research InstitutionKyoto University

Principal Investigator

Kojima Yusuke  京都大学, iPS細胞研究所, 特定拠点助教 (90896416)

Project Period (FY) 2021-04-01 – 2024-03-31
Project Status Completed (Fiscal Year 2023)
Budget Amount *help
¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2023: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2022: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2021: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Keywordsゲノム編集 / CRISPR-Cas3 / CRISPR-Cas / 遺伝子ノックアウト / タンパク質障害物 / 遺伝子疾患 / 遺伝子治療
Outline of Research at the Start

遺伝子配列を編集する技術の開発は、生物の基礎研究や遺伝子疾患の治療などにおいて重要である。近年、CRISPR-Cas3を用いた新しいゲノム編集技術が開発されたが、長距離のゲノム切断を行えるという、他の酵素には無い特徴を持つ一方で、その長さを制御する方法はまだ確立されていない。そこで本研究では、Cas3による遺伝子変異を制御する方法の開発を行う。

Outline of Final Research Achievements

In this project, we aimed to regulate CRISPR-Cas3 deletion length by using protein obstacles, and also, to establish a method to analyze collisions between Cas3 and other proteins on the genome. As its achievements, we could obtain many useful insights into Cas3 deletion patterns although practical and easy Cas3 regulation methods have not been established within this period. We also could develop an improved method to detect protein-protein collisions on the genome. Then, eventually, we could publish one research paper and submit one patent containing insights obtained from this project.

Academic Significance and Societal Importance of the Research Achievements

本課題を通じて、新規国産ゲノム編集技術であるCRISPR-Cas3について、細胞内の動態をより理解でき、欠損領域をどのように制御するかという課題に対して多くの知見を得ることができた。これら得られた知見は、将来的にCas3を有用なゲノム編集技術として確立し、特に遺伝子疾患などの難病をゲノム編集や再生医療の技術を用いた治療に応用する上で非常に意義のあることであったと考える。

Report

(4 results)
  • 2023 Annual Research Report   Final Research Report ( PDF )
  • 2022 Research-status Report
  • 2021 Research-status Report
  • Research Products

    (2 results)

All 2024 2023

All Journal Article (1 results) (of which Peer Reviewed: 1 results,  Open Access: 1 results) Patent(Industrial Property Rights) (1 results) (of which Overseas: 1 results)

  • [Journal Article] Dual CRISPR-Cas3 system for inducing multi-exon skipping in DMD patient-derived iPSCs2023

    • Author(s)
      Kita Yuto、Okuzaki Yuya、Naoe Youichi、Lee Joseph、Bang Uikyu、Okawa Natsumi、Ichiki Akane、Jonouchi Tatsuya、Sakurai Hidetoshi、Kojima Yusuke、Hotta Akitsu
    • Journal Title

      Stem Cell Reports

      Volume: 18 Issue: 9 Pages: 1753-1765

    • DOI

      10.1016/j.stemcr.2023.07.007

    • Related Report
      2023 Annual Research Report
    • Peer Reviewed / Open Access
  • [Patent(Industrial Property Rights)] 修飾ヌクレオチドを有するcrRNA2024

    • Inventor(s)
      堀田秋津、小島佑介、北悠人
    • Industrial Property Rights Holder
      堀田秋津、小島佑介、北悠人
    • Industrial Property Rights Type
      特許
    • Filing Date
      2024
    • Related Report
      2023 Annual Research Report
    • Overseas

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Published: 2021-04-28   Modified: 2025-01-30  

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