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Development of genome editing platform technology for various disease models

Research Project

Project/Area Number 17H01409
Research Category

Grant-in-Aid for Scientific Research (A)

Allocation TypeSingle-year Grants
Section一般
Research Field Medical genome science
Research InstitutionHiroshima University

Principal Investigator

YAMAMOTO TAKASHI  広島大学, 統合生命科学研究科(理), 教授 (90244102)

Co-Investigator(Kenkyū-buntansha) 佐久間 哲史  広島大学, 統合生命科学研究科(理), 准教授 (90711143)
細羽 康介  広島大学, 統合生命科学研究科(理), 助教 (20781264)
Project Period (FY) 2017-04-01 – 2021-03-31
Project Status Completed (Fiscal Year 2020)
Budget Amount *help
¥41,990,000 (Direct Cost: ¥32,300,000、Indirect Cost: ¥9,690,000)
Fiscal Year 2020: ¥9,100,000 (Direct Cost: ¥7,000,000、Indirect Cost: ¥2,100,000)
Fiscal Year 2019: ¥9,100,000 (Direct Cost: ¥7,000,000、Indirect Cost: ¥2,100,000)
Fiscal Year 2018: ¥9,100,000 (Direct Cost: ¥7,000,000、Indirect Cost: ¥2,100,000)
Fiscal Year 2017: ¥14,690,000 (Direct Cost: ¥11,300,000、Indirect Cost: ¥3,390,000)
Keywordsゲノム編集 / 疾患モデル / 疾患モデル細胞 / SNP / バイオテクノロジー
Outline of Final Research Achievements

The purpose of this study is to develop platform technology for generating disease model cultured cells and animals. Based on the PITCh (Precise integration of target chromosome) method for insertion of exogenous DNA into multiple target loci that we have reported, I developed LoAD (Local accumulation of DSB molecules) system for enhancing accumulation of DSB molecules). I also developed a program of NGS data analysis (Machiato) for evaluating knock-in accuracy and calculated the efficiency of accurate knock-in and NHEJ indel mutations on human 40 gene loci. Furthermore, I made knock-in っcells and mice harboring disease SNPs of mucopolysaccharidosis.

Academic Significance and Societal Importance of the Research Achievements

本研究は、様々な生物において正確に遺伝子を改変することができるゲノム編集技術について複数の新しい技術と解析法を開発し、正確な遺伝子組換えや疾患モデルを作製することに成功した。これらの技術によって、様々な疾患のモデルを作製することが可能となり、疾患の発症メカニズムの解明、疾患の治療法の開発、再生医療における新しい技術の提供が可能になることが期待される。

Report

(5 results)
  • 2020 Annual Research Report   Final Research Report ( PDF )
  • 2019 Annual Research Report
  • 2018 Annual Research Report
  • 2017 Annual Research Report
  • Research Products

    (27 results)

All 2021 2020 2019 2018 2017 Other

All Int'l Joint Research (2 results) Journal Article (11 results) (of which Int'l Joint Research: 4 results,  Peer Reviewed: 11 results,  Open Access: 11 results,  Acknowledgement Compliant: 1 results) Presentation (8 results) (of which Int'l Joint Research: 3 results,  Invited: 7 results) Book (3 results) Remarks (2 results) Patent(Industrial Property Rights) (1 results)

  • [Int'l Joint Research] University of Liverpool(英国)

    • Related Report
      2020 Annual Research Report
  • [Int'l Joint Research] リバプール大学(英国)

    • Related Report
      2018 Annual Research Report
  • [Journal Article] Genome-wide kinetic properties of transcriptional bursting in mouse embryonic stem cells2020

    • Author(s)
      Ochiai Hiroshi、Hayashi Tetsutaro、Umeda Mana、Yoshimura Mika、Harada Akihito、Shimizu Yukiko、Nakano Kenta、Saitoh Noriko、Liu Zhe、Yamamoto Takashi、Okamura Tadashi、Ohkawa Yasuyuki、Kimura Hiroshi、Nikaido Itoshi
    • Journal Title

      Science Advances

      Volume: 6 Issue: 25

    • DOI

      10.1126/sciadv.aaz6699

    • Related Report
      2020 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Insufficiency of ciliary cholesterol in hereditary Zellweger syndrome.2020

    • Author(s)
      Miyamoto T, Hosoba K, Itabashi T, Iwane AH, Akutsu AN, Ochiai H, Saito Y, Yamamoto T, Matsuura S
    • Journal Title

      EMBO Journal

      Volume: - Issue: 12

    • DOI

      10.15252/embj.2019103499

    • Related Report
      2020 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Single-gene imaging links genome topology, promoter?enhancer communication and transcription control2020

    • Author(s)
      Li Jieru、Hsu Angela、Hua Yujing、Wang Guanshi、Cheng Lingling、Ochiai Hiroshi、Yamamoto Takashi、Pertsinidis Alexandros
    • Journal Title

      Nature Structural & Molecular Biology

      Volume: 27 Issue: 11 Pages: 1032-1040

    • DOI

      10.1038/s41594-020-0493-6

    • Related Report
      2020 Annual Research Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Anephrogenic phenotype induced by SALL1 gene knockout in pigs.2019

    • Author(s)
      Watanabe M, Nakano K, Uchikura A, Matsunari H, Yashima S, Umeyama K, Takayanagi S, Sakuma T, Yamamoto T, Morita S, Horii T, Hatada I, Nishinakamura R, Nakauchi H, Nagashima H.
    • Journal Title

      Sci Rep.

      Volume: 9 Issue: 1 Pages: 8016-8016

    • DOI

      10.1038/s41598-019-44387-w

    • Related Report
      2019 Annual Research Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Single-Molecule Nanoscopy Elucidates RNA Polymerase II Transcription at Single Genes in Live Cells2019

    • Author(s)
      Li Jieru、Dong Ankun、Saydaminova Kamola、Chang Hill、Wang Guanshi、Ochiai Hiroshi、Yamamoto Takashi、Pertsinidis Alexandros
    • Journal Title

      Cell

      Volume: 178 Issue: 2 Pages: 491-506

    • DOI

      10.1016/j.cell.2019.05.029

    • Related Report
      2019 Annual Research Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Activin Is Superior to BMP7 for Efficient Maintenance of Human iPSC-Derived Nephron Progenitors.2019

    • Author(s)
      Tanigawa S, Naganuma H, Kaku Y, Era T, Sakuma T, Yamamoto T, Taguchi A and Nishinakamura R.
    • Journal Title

      Stem Cell Reports

      Volume: 13 Issue: 2 Pages: 322-337

    • DOI

      10.1016/j.stemcr.2019.07.003

    • Related Report
      2019 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] CRISPR-Cas3 induces broad and unidirectional genome editing in human cells2019

    • Author(s)
      Morisaka Hiroyuki、Yoshimi Kazuto、Okuzaki Yuya、Gee Peter、Kunihiro Yayoi、Sonpho Ekasit、Xu Huaigeng、Sasakawa Noriko、Naito Yuki、Nakada Shinichiro、Yamamoto Takashi、Sano Shigetoshi、Hotta Akitsu、Takeda Junji、Mashimo Tomoji
    • Journal Title

      Nature Communications

      Volume: 10 Issue: 1 Pages: 5302-5302

    • DOI

      10.1038/s41467-019-13226-x

    • NAID

      120006848247

    • Related Report
      2019 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Biased genome editing using the local accumulation of DSB repair molecules system.2018

    • Author(s)
      Nakade S, Mochida K, Kunii A, Nakamae K, Aida T, Tanaka K, Sakamoto N, Sakuma T, Yamamoto T
    • Journal Title

      Nature Communications

      Volume: 9(1) Issue: 1 Pages: 3270-3270

    • DOI

      10.1038/s41467-018-05773-6

    • Related Report
      2018 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Three-Component Repurposed Technology for Enhanced Expression: Highly Accumulable Transcriptional Activators via Branched Tag Arrays2018

    • Author(s)
      Kunii A, Hara Y, Takenaga M, Hattori N, Fukazawa T, Ushijima T, Yamamoto T, Sakuma T.
    • Journal Title

      CRISPR J

      Volume: 1 Issue: 5 Pages: 337-347

    • DOI

      10.1089/crispr.2018.0009

    • Related Report
      2018 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Microhomology-assisted scarless genome editing in human iPSCs2018

    • Author(s)
      Kim Shin-Il、Matsumoto Tomoko、Kagawa Harunobu、Nakamura Michiko、Hirohata Ryoko、Ueno Ayano、Ohishi Maki、Sakuma Tetsushi、Soga Tomoyoshi、Yamamoto Takashi、Woltjen Knut
    • Journal Title

      Nature Communications

      Volume: 9 Issue: 1 Pages: 939-939

    • DOI

      10.1038/s41467-018-03044-y

    • Related Report
      2017 Annual Research Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Establishment of expanded and streamlined pipeline of PITCh knock-in - a web-based design tool for MMEJ-mediated gene knock-in, PITCh designer, and the variations of PITCh, PITCh-TG and PITCh-KIKO2017

    • Author(s)
      Nakamae K, Nishimura Y, Takenaga M, Nakade S, Sakamoto N, Ide H, Sakuma T, Yamamoto T
    • Journal Title

      Bioengineered

      Volume: - Issue: 3 Pages: 302-308

    • DOI

      10.1080/21655979.2017.1313645

    • Related Report
      2017 Annual Research Report
    • Peer Reviewed / Open Access / Acknowledgement Compliant
  • [Presentation] ゲノム編集に関する最近の研究動向2021

    • Author(s)
      山本 卓
    • Organizer
      日本薬学会 第141年会
    • Related Report
      2020 Annual Research Report
    • Invited
  • [Presentation] ゲノム編集技術の最前線2020

    • Author(s)
      山本 卓
    • Organizer
      第7回京都大学-稲盛財団合同「京都賞シンポジウム」
    • Related Report
      2020 Annual Research Report
    • Invited
  • [Presentation] ゲノム編集医療の可能性2020

    • Author(s)
      山本 卓
    • Organizer
      第65回日本生殖医学会学術講演会・総会
    • Related Report
      2020 Annual Research Report
    • Invited
  • [Presentation] Applications of Genome Editing Technology for Various Life Science Research2019

    • Author(s)
      Yamamoto T
    • Organizer
      GWG 2019 Genome Engineering Conference
    • Related Report
      2019 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] Concurrent MMEJ-assisted fusional knock-in of long gene cassette in human cells and mouse embryos2018

    • Author(s)
      Sakuma T et al.
    • Organizer
      Keystone Symposia Conference _ Genome Engineering
    • Related Report
      2018 Annual Research Report
    • Int'l Joint Research
  • [Presentation] Basics of genome editing and biased genome editing using the LoAD system in cultured cells and organisms2018

    • Author(s)
      Yamamoto T
    • Organizer
      Functional Genomics and Structural Biology (FGSB)
    • Related Report
      2018 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] Advances in genome editing technology and its applications in biomedical fields2017

    • Author(s)
      Takashi Yamamoto
    • Organizer
      The 23rd Annual Meeting of Japan Society of Gene and Cell Therapy
    • Related Report
      2017 Annual Research Report
    • Invited
  • [Presentation] CRISPR-Cas9の再生医療での可能性2017

    • Author(s)
      山本 卓
    • Organizer
      第2回再生医療産学官連携シンポジウム
    • Related Report
      2017 Annual Research Report
    • Invited
  • [Book] ゲノム編集とはなにか2020

    • Author(s)
      山本 卓
    • Total Pages
      240
    • Publisher
      講談社
    • ISBN
      9784065194690
    • Related Report
      2020 Annual Research Report
  • [Book] 完全版 ゲノム編集実験スタンダード2019

    • Author(s)
      山本 卓、佐久間哲史
    • Total Pages
      386
    • Publisher
      羊土社
    • ISBN
      9784758122443
    • Related Report
      2019 Annual Research Report
  • [Book] ゲノム編集の基本原理と応用2018

    • Author(s)
      山本 卓
    • Total Pages
      176
    • Publisher
      裳華房
    • ISBN
      9784785358693
    • Related Report
      2018 Annual Research Report
  • [Remarks] 分子遺伝学研究室

    • URL

      https://www.mls.sci.hiroshima-u.ac.jp/smg/index.html

    • Related Report
      2020 Annual Research Report
  • [Remarks] 分子遺伝学研究室

    • URL

      http://www.mls.sci.hiroshima-u.ac.jp/smg/index.html

    • Related Report
      2019 Annual Research Report 2018 Annual Research Report
  • [Patent(Industrial Property Rights)] 標的遺伝子にエフェクタータンパク質を集積するための組成物、およびその利用2018

    • Inventor(s)
      山本卓、佐久間哲史、國井厚志
    • Industrial Property Rights Holder
      山本卓、佐久間哲史、國井厚志
    • Industrial Property Rights Type
      特許
    • Filing Date
      2018
    • Related Report
      2018 Annual Research Report

URL: 

Published: 2017-04-28   Modified: 2022-01-27  

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