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In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

Author

Listed:
  • Keiichiro Suzuki

    (Gene Expression Laboratory, Salk Institute for Biological Studies)

  • Yuji Tsunekawa

    (Laboratory for Cell Asymmetry, RIKEN Center for Developmental Biology)

  • Reyna Hernandez-Benitez

    (Gene Expression Laboratory, Salk Institute for Biological Studies
    4700 King Abdullah University of Science and Technology (KAUST))

  • Jun Wu

    (Gene Expression Laboratory, Salk Institute for Biological Studies
    Universidad Católica San Antonio de Murcia (UCAM) Campus de los Jerónimos)

  • Jie Zhu

    (Guangzhou Women and Children’s Medical Center, Guangzhou Medical University
    Shiley Eye Institute, Institute for Genomic Medicine, Institute of Engineering in Medicine, University of California, San Diego)

  • Euiseok J. Kim

    (Systems Neurobiology Laboratory, Salk Institute for Biological Studies)

  • Fumiyuki Hatanaka

    (Gene Expression Laboratory, Salk Institute for Biological Studies)

  • Mako Yamamoto

    (Gene Expression Laboratory, Salk Institute for Biological Studies)

  • Toshikazu Araoka

    (Gene Expression Laboratory, Salk Institute for Biological Studies
    Universidad Católica San Antonio de Murcia (UCAM) Campus de los Jerónimos)

  • Zhe Li

    (Bioengineering, University of California, San Diego)

  • Masakazu Kurita

    (Gene Expression Laboratory, Salk Institute for Biological Studies)

  • Tomoaki Hishida

    (Gene Expression Laboratory, Salk Institute for Biological Studies)

  • Mo Li

    (Gene Expression Laboratory, Salk Institute for Biological Studies)

  • Emi Aizawa

    (Gene Expression Laboratory, Salk Institute for Biological Studies)

  • Shicheng Guo

    (Bioengineering, University of California, San Diego)

  • Song Chen

    (Bioengineering, University of California, San Diego)

  • April Goebl

    (Gene Expression Laboratory, Salk Institute for Biological Studies)

  • Rupa Devi Soligalla

    (Gene Expression Laboratory, Salk Institute for Biological Studies)

  • Jing Qu

    (State Key Laboratory of Stem Cell and Reproductive Biology, Institute of Zoology, Chinese Academy of Sciences
    University of Chinese Academy of Sciences)

  • Tingshuai Jiang

    (Shiley Eye Institute, Institute for Genomic Medicine, Institute of Engineering in Medicine, University of California, San Diego
    Guangzhou EliteHealth Biological Pharmaceutical Technology Company Ltd)

  • Xin Fu

    (Guangzhou Women and Children’s Medical Center, Guangzhou Medical University
    Shiley Eye Institute, Institute for Genomic Medicine, Institute of Engineering in Medicine, University of California, San Diego)

  • Maryam Jafari

    (Shiley Eye Institute, Institute for Genomic Medicine, Institute of Engineering in Medicine, University of California, San Diego)

  • Concepcion Rodriguez Esteban

    (Gene Expression Laboratory, Salk Institute for Biological Studies)

  • W. Travis Berggren

    (Salk Institute for Biological Studies)

  • Jeronimo Lajara

    (Universidad Católica San Antonio de Murcia (UCAM) Campus de los Jerónimos)

  • Estrella Nuñez-Delicado

    (Universidad Católica San Antonio de Murcia (UCAM) Campus de los Jerónimos)

  • Pedro Guillen

    (Universidad Católica San Antonio de Murcia (UCAM) Campus de los Jerónimos
    Fundación Dr. Pedro Guillen, Investigación Biomedica de Clinica CEMTRO)

  • Josep M. Campistol

    (Hospital Clinic, University of Barcelona, IDIBAPS)

  • Fumio Matsuzaki

    (Laboratory for Cell Asymmetry, RIKEN Center for Developmental Biology)

  • Guang-Hui Liu

    (University of Chinese Academy of Sciences
    National Laboratory of Biomacromolecules, CAS Center for Excellence in Biomacromolecules, Institute of Biophysics, Chinese Academy of Sciences
    Key Laboratory of Regenerative Medicine of Ministry of Education, Institute of Aging and Regenerative Medicine, Jinan University
    Beijing Institute for Brain Disorders)

  • Pierre Magistretti

    (4700 King Abdullah University of Science and Technology (KAUST))

  • Kun Zhang

    (Bioengineering, University of California, San Diego)

  • Edward M. Callaway

    (Systems Neurobiology Laboratory, Salk Institute for Biological Studies)

  • Kang Zhang

    (Guangzhou Women and Children’s Medical Center, Guangzhou Medical University
    Shiley Eye Institute, Institute for Genomic Medicine, Institute of Engineering in Medicine, University of California, San Diego
    Molecular Medicine Research Center, West China Hospital, Sichuan University
    Veterans Administration Healthcare System)

  • Juan Carlos Izpisua Belmonte

    (Gene Expression Laboratory, Salk Institute for Biological Studies)

Abstract

A method for CRISPR-based genome editing that harnesses cellular non-homologous end joining activity to achieve targeted DNA knock-in in non-dividing tissues.

Suggested Citation

  • Keiichiro Suzuki & Yuji Tsunekawa & Reyna Hernandez-Benitez & Jun Wu & Jie Zhu & Euiseok J. Kim & Fumiyuki Hatanaka & Mako Yamamoto & Toshikazu Araoka & Zhe Li & Masakazu Kurita & Tomoaki Hishida & Mo, 2016. "In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration," Nature, Nature, vol. 540(7631), pages 144-149, December.
    • Handle: RePEc:nat:nature:v:540:y:2016:i:7631:d:10.1038_nature20565
    DOI: 10.1038/nature20565
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    Citations

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    Cited by:

    1. Sandra Wimberger & Nina Akrap & Mike Firth & Johan Brengdahl & Susanna Engberg & Marie K. Schwinn & Michael R. Slater & Anders Lundin & Pei-Pei Hsieh & Songyuan Li & Silvia Cerboni & Jonathan Sumner &, 2023. "Simultaneous inhibition of DNA-PK and Polϴ improves integration efficiency and precision of genome editing," Nature Communications, Nature, vol. 14(1), pages 1-18, December.
    2. Takeshi Hori & Hiroaki Okae & Shun Shibata & Norio Kobayashi & Eri H. Kobayashi & Akira Oike & Asato Sekiya & Takahiro Arima & Hirokazu Kaji, 2024. "Trophoblast stem cell-based organoid models of the human placental barrier," Nature Communications, Nature, vol. 15(1), pages 1-15, December.
    3. Jun Huang & David Rowe & Pratima Subedi & Wei Zhang & Tyler Suelter & Barbara Valent & David E. Cook, 2022. "CRISPR-Cas12a induced DNA double-strand breaks are repaired by multiple pathways with different mutation profiles in Magnaporthe oryzae," Nature Communications, Nature, vol. 13(1), pages 1-18, December.
    4. Patricia R. Pitrez & Luis M. Monteiro & Oliver Borgogno & Xavier Nissan & Jerome Mertens & Lino Ferreira, 2024. "Cellular reprogramming as a tool to model human aging in a dish," Nature Communications, Nature, vol. 15(1), pages 1-11, December.
    5. Ryoko Kuwayama & Keiichiro Suzuki & Jun Nakamura & Emi Aizawa & Yoshichika Yoshioka & Masahito Ikawa & Shin Nabatame & Ken-ichi Inoue & Yoshiari Shimmyo & Keiichi Ozono & Taroh Kinoshita & Yoshiko Mur, 2022. "Establishment of mouse model of inherited PIGO deficiency and therapeutic potential of AAV-based gene therapy," Nature Communications, Nature, vol. 13(1), pages 1-16, December.
    6. Patrizia Tornabene & Rita Ferla & Manel Llado-Santaeularia & Miriam Centrulo & Margherita Dell’Anno & Federica Esposito & Elena Marrocco & Emanuela Pone & Renato Minopoli & Carolina Iodice & Edoardo N, 2022. "Therapeutic homology-independent targeted integration in retina and liver," Nature Communications, Nature, vol. 13(1), pages 1-14, December.
    7. Raed Ibraheim & Phillip W. L. Tai & Aamir Mir & Nida Javeed & Jiaming Wang & Tomás C. Rodríguez & Suk Namkung & Samantha Nelson & Eraj Shafiq Khokhar & Esther Mintzer & Stacy Maitland & Zexiang Chen &, 2021. "Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo," Nature Communications, Nature, vol. 12(1), pages 1-17, December.
    8. Xiangjun He & Zhenjie Zhang & Junyi Xue & Yaofeng Wang & Siqi Zhang & Junkang Wei & Chenzi Zhang & Jue Wang & Brian Anugerah Urip & Chun Christopher Ngan & Junjiang Sun & Yuefeng Li & Zhiqian Lu & Hui, 2022. "Low-dose AAV-CRISPR-mediated liver-specific knock-in restored hemostasis in neonatal hemophilia B mice with subtle antibody response," Nature Communications, Nature, vol. 13(1), pages 1-17, December.
    9. Zengpeng Han & Nengsong Luo & Wenyu Ma & Xiaodong Liu & Yuxiang Cai & Jiaxin Kou & Jie Wang & Lei Li & Siqi Peng & Zihong Xu & Wen Zhang & Yuxiang Qiu & Yang Wu & Chaohui Ye & Kunzhang Lin & Fuqiang X, 2023. "AAV11 enables efficient retrograde targeting of projection neurons and enhances astrocyte-directed transduction," Nature Communications, Nature, vol. 14(1), pages 1-13, December.
    10. Xiaoguang Pan & Kunli Qu & Hao Yuan & Xi Xiang & Christian Anthon & Liubov Pashkova & Xue Liang & Peng Han & Giulia I. Corsi & Fengping Xu & Ping Liu & Jiayan Zhong & Yan Zhou & Tao Ma & Hui Jiang & J, 2022. "Massively targeted evaluation of therapeutic CRISPR off-targets in cells," Nature Communications, Nature, vol. 13(1), pages 1-14, December.
    11. Yuki Ogawa & Brian C. Lim & Shanu George & Juan A. Oses-Prieto & Joshua M. Rasband & Yael Eshed-Eisenbach & Hamdan Hamdan & Supna Nair & Francesco Boato & Elior Peles & Alma L. Burlingame & Linda Aels, 2023. "Antibody-directed extracellular proximity biotinylation reveals that Contactin-1 regulates axo-axonic innervation of axon initial segments," Nature Communications, Nature, vol. 14(1), pages 1-18, December.
    12. Maria Pallarès-Masmitjà & Dimitrije Ivančić & Júlia Mir-Pedrol & Jessica Jaraba-Wallace & Tommaso Tagliani & Baldomero Oliva & Amal Rahmeh & Avencia Sánchez-Mejías & Marc Güell, 2021. "Find and cut-and-transfer (FiCAT) mammalian genome engineering," Nature Communications, Nature, vol. 12(1), pages 1-9, December.
    13. Martin Peterka & Nina Akrap & Songyuan Li & Sandra Wimberger & Pei-Pei Hsieh & Dmitrii Degtev & Burcu Bestas & Jack Barr & Stijn Plassche & Patricia Mendoza-Garcia & Saša Šviković & Grzegorz Sienski &, 2022. "Harnessing DSB repair to promote efficient homology-dependent and -independent prime editing," Nature Communications, Nature, vol. 13(1), pages 1-9, December.
    14. Matteo Ciciani & Michele Demozzi & Eleonora Pedrazzoli & Elisabetta Visentin & Laura Pezzè & Lorenzo Federico Signorini & Aitor Blanco-Miguez & Moreno Zolfo & Francesco Asnicar & Antonio Casini & Anna, 2022. "Automated identification of sequence-tailored Cas9 proteins using massive metagenomic data," Nature Communications, Nature, vol. 13(1), pages 1-8, December.
    15. Akiko Tomita & Hiroyuki Sasanuma & Tomoo Owa & Yuka Nakazawa & Mayuko Shimada & Takahiro Fukuoka & Tomoo Ogi & Shinichiro Nakada, 2023. "Inducing multiple nicks promotes interhomolog homologous recombination to correct heterozygous mutations in somatic cells," Nature Communications, Nature, vol. 14(1), pages 1-20, December.

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