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Extension of the crRNA enhances Cpf1 gene editing in vitro and in vivo

Author

Listed:
  • Hyo Min Park

    (GenEdit Inc.)

  • Hui Liu

    (GenEdit Inc.)

  • Joann Wu

    (GenEdit Inc.)

  • Anthony Chong

    (GenEdit Inc.)

  • Vanessa Mackley

    (GenEdit Inc.)

  • Christof Fellmann

    (University of California, Berkeley)

  • Anirudh Rao

    (University of California, Berkeley)

  • Fuguo Jiang

    (University of California, Berkeley)

  • Hunghao Chu

    (GenEdit Inc.)

  • Niren Murthy

    (University of California, Berkeley)

  • Kunwoo Lee

    (GenEdit Inc.)

Abstract

Engineering of the Cpf1 crRNA has the potential to enhance its gene editing efficiency and non-viral delivery to cells. Here, we demonstrate that extending the length of its crRNA at the 5′ end can enhance the gene editing efficiency of Cpf1 both in cells and in vivo. Extending the 5′ end of the crRNA enhances the gene editing efficiency of the Cpf1 RNP to induce non-homologous end-joining and homology-directed repair using electroporation in cells. Additionally, chemical modifications on the extended 5′ end of the crRNA result in enhanced serum stability. Also, extending the 5′ end of the crRNA by 59 nucleotides increases the delivery efficiency of Cpf1 RNP in cells and in vivo cationic delivery vehicles including polymer nanoparticle. Thus, 5′ extension and chemical modification of the Cpf1 crRNA is an effective method for enhancing the gene editing efficiency of Cpf1 and its delivery in vivo.

Suggested Citation

  • Hyo Min Park & Hui Liu & Joann Wu & Anthony Chong & Vanessa Mackley & Christof Fellmann & Anirudh Rao & Fuguo Jiang & Hunghao Chu & Niren Murthy & Kunwoo Lee, 2018. "Extension of the crRNA enhances Cpf1 gene editing in vitro and in vivo," Nature Communications, Nature, vol. 9(1), pages 1-12, December.
    • Handle: RePEc:nat:natcom:v:9:y:2018:i:1:d:10.1038_s41467-018-05641-3
    DOI: 10.1038/s41467-018-05641-3
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    Cited by:

    1. Eman A. Ageely & Ramadevi Chilamkurthy & Sunit Jana & Leonora Abdullahu & Daniel O’Reilly & Philip J. Jensik & Masad J. Damha & Keith T. Gagnon, 2021. "Gene editing with CRISPR-Cas12a guides possessing ribose-modified pseudoknot handles," Nature Communications, Nature, vol. 12(1), pages 1-15, December.

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