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Cas9 immunity creates challenges for CRISPR gene editing therapies

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  • Julie M. Crudele

    (University of Washington
    University of Washington)

  • Jeffrey S. Chamberlain

    (University of Washington
    University of Washington)

Abstract

Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 is a genome-editing technology1,2 that utilizes archaeal and bacterial Cas9 nucleases to introduce double-stranded breaks in DNA at targeted sites. These breaks can be used to remove, replace, or add pieces of DNA. While not the first genome editor, CRISPR-Cas9 is efficient and cost-effective because cutting is guided by a strand of RNA rather than a protein. The potential uses in health care are plentiful, from disrupting dominant genes that cause cancer3 to repairing mutated genes that cause genetic diseases, such as muscular dystrophy4. Therapeutic approaches based on this technology fill the preclinical pipeline, and rely on the use of viral vectors to deliver the Cas9 gene and guide RNA to a gene of interest. However, concerns regarding the safety and efficacy of CRISPR-Cas9 use in gene therapy remain. A pre-print released prior to peer review has recently underlined the question of whether immunological responses to Cas9 may negatively impact its clinical use5. Here we discuss the implications of this finding for the application of CRISPR/Cas in gene therapy.

Suggested Citation

  • Julie M. Crudele & Jeffrey S. Chamberlain, 2018. "Cas9 immunity creates challenges for CRISPR gene editing therapies," Nature Communications, Nature, vol. 9(1), pages 1-3, December.
    • Handle: RePEc:nat:natcom:v:9:y:2018:i:1:d:10.1038_s41467-018-05843-9
    DOI: 10.1038/s41467-018-05843-9
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    Cited by:

    1. Zhangyi Luo & Yixian Huang & Neelu Batra & Yuang Chen & Haozhe Huang & Yifei Wang & Ziqian Zhang & Shichen Li & Chien-Yu Chen & Zehua Wang & Jingjing Sun & Qiming Jane Wang & Da Yang & Binfeng Lu & Ja, 2024. "Inhibition of iRhom1 by CD44-targeting nanocarrier for improved cancer immunochemotherapy," Nature Communications, Nature, vol. 15(1), pages 1-20, December.
    2. Chady H. Hakim & Sandeep R. P. Kumar & Dennis O. Pérez-López & Nalinda B. Wasala & Dong Zhang & Yongping Yue & James Teixeira & Xiufang Pan & Keqing Zhang & Emily D. Million & Christopher E. Nelson & , 2021. "Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models," Nature Communications, Nature, vol. 12(1), pages 1-12, December.
    3. Qiao Liu & Di He & Lei Xie, 2019. "Prediction of off-target specificity and cell-specific fitness of CRISPR-Cas System using attention boosted deep learning and network-based gene feature," PLOS Computational Biology, Public Library of Science, vol. 15(10), pages 1-22, October.
    4. Marcus A. Toral & Carsten T. Charlesworth & Benjamin Ng & Teja Chemudupati & Shota Homma & Hiromitsu Nakauchi & Alexander G. Bassuk & Matthew H. Porteus & Vinit B. Mahajan, 2022. "Investigation of Cas9 antibodies in the human eye," Nature Communications, Nature, vol. 13(1), pages 1-9, December.

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