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The promise and challenge of therapeutic genome editing

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  • Jennifer A. Doudna

    (University of California Berkeley
    University of California Berkeley
    University of California Berkeley
    University of California Berkeley)

Abstract

Genome editing, which involves the precise manipulation of cellular DNA sequences to alter cell fates and organism traits, has the potential to both improve our understanding of human genetics and cure genetic disease. Here I discuss the scientific, technical and ethical aspects of using CRISPR (clustered regularly interspaced short palindromic repeats) technology for therapeutic applications in humans, focusing on specific examples that highlight both opportunities and challenges. Genome editing is—or will soon be—in the clinic for several diseases, with more applications under development. The rapid pace of the field demands active efforts to ensure that this breakthrough technology is used responsibly to treat, cure and prevent genetic disease.

Suggested Citation

  • Jennifer A. Doudna, 2020. "The promise and challenge of therapeutic genome editing," Nature, Nature, vol. 578(7794), pages 229-236, February.
    • Handle: RePEc:nat:nature:v:578:y:2020:i:7794:d:10.1038_s41586-020-1978-5
    DOI: 10.1038/s41586-020-1978-5
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    Cited by:

    1. Eleonora Pedrazzoli & Michele Demozzi & Elisabetta Visentin & Matteo Ciciani & Ilaria Bonuzzi & Laura Pezzè & Lorenzo Lucchetta & Giulia Maule & Simone Amistadi & Federica Esposito & Mariangela Lupo &, 2024. "CoCas9 is a compact nuclease from the human microbiome for efficient and precise genome editing," Nature Communications, Nature, vol. 15(1), pages 1-12, December.
    2. Bert van de Kooij & Alex Kruswick & Haico van Attikum & Michael B. Yaffe, 2022. "Multi-pathway DNA-repair reporters reveal competition between end-joining, single-strand annealing and homologous recombination at Cas9-induced DNA double-strand breaks," Nature Communications, Nature, vol. 13(1), pages 1-17, December.
    3. Francisco Tenjo-Castaño & Nicholas Sofos & Blanca López-Méndez & Luisa S. Stutzke & Anders Fuglsang & Stefano Stella & Guillermo Montoya, 2022. "Structure of the TnsB transposase-DNA complex of type V-K CRISPR-associated transposon," Nature Communications, Nature, vol. 13(1), pages 1-14, December.
    4. 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.
    5. 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.
    6. Meiling Lu & Chenlin Yu & Yuwen Zhang & Wenjun Ju & Zhi Ye & Chenyang Hua & Jinze Mao & Chunyi Hu & Zhenhuang Yang & Yibei Xiao, 2024. "Structure and genome editing of type I-B CRISPR-Cas," Nature Communications, Nature, vol. 15(1), pages 1-12, December.
    7. Changchang Xin & Jianhang Yin & Shaopeng Yuan & Liqiong Ou & Mengzhu Liu & Weiwei Zhang & Jiazhi Hu, 2022. "Comprehensive assessment of miniature CRISPR-Cas12f nucleases for gene disruption," Nature Communications, Nature, vol. 13(1), pages 1-10, December.
    8. Xiangfeng Kong & Hainan Zhang & Guoling Li & Zikang Wang & Xuqiang Kong & Lecong Wang & Mingxing Xue & Weihong Zhang & Yao Wang & Jiajia Lin & Jingxing Zhou & Xiaowen Shen & Yinghui Wei & Na Zhong & W, 2023. "Engineered CRISPR-OsCas12f1 and RhCas12f1 with robust activities and expanded target range for genome editing," Nature Communications, Nature, vol. 14(1), pages 1-13, December.
    9. Kai Chen & Elizabeth C. Stahl & Min Hyung Kang & Bryant Xu & Ryan Allen & Marena Trinidad & Jennifer A. Doudna, 2024. "Engineering self-deliverable ribonucleoproteins for genome editing in the brain," Nature Communications, Nature, vol. 15(1), pages 1-11, December.
    10. Xiangyang Li & Guiquan Zhang & Shisheng Huang & Yao Liu & Jin Tang & Mingtian Zhong & Xin Wang & Wenjun Sun & Yuan Yao & Quanjiang Ji & Xiaolong Wang & Jianghuai Liu & Shiqiang Zhu & Xingxu Huang, 2023. "Development of a versatile nuclease prime editor with upgraded precision," Nature Communications, Nature, vol. 14(1), pages 1-13, December.
    11. Juan A. Perez-Bermejo & Oghene Efagene & William M. Matern & Jeffrey K. Holden & Shaheen Kabir & Glen M. Chew & Gaia Andreoletti & Eniola Catton & Craig L. Ennis & Angelica Garcia & Trevor L. Gerstenb, 2024. "Functional screening in human HSPCs identifies optimized protein-based enhancers of Homology Directed Repair," Nature Communications, Nature, vol. 15(1), pages 1-16, December.
    12. Aakash Saha & Mohd Ahsan & Pablo R. Arantes & Michael Schmitz & Christelle Chanez & Martin Jinek & Giulia Palermo, 2024. "An alpha-helical lid guides the target DNA toward catalysis in CRISPR-Cas12a," Nature Communications, Nature, vol. 15(1), pages 1-12, December.
    13. Jianli Tao & Daniel E. Bauer & Roberto Chiarle, 2023. "Assessing and advancing the safety of CRISPR-Cas tools: from DNA to RNA editing," Nature Communications, Nature, vol. 14(1), pages 1-16, December.
    14. Péter István Kulcsár & András Tálas & Zoltán Ligeti & Eszter Tóth & Zsófia Rakvács & Zsuzsa Bartos & Sarah Laura Krausz & Ágnes Welker & Vanessza Laura Végi & Krisztina Huszár & Ervin Welker, 2023. "A cleavage rule for selection of increased-fidelity SpCas9 variants with high efficiency and no detectable off-targets," Nature Communications, Nature, vol. 14(1), pages 1-20, December.
    15. 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.
    16. Burcu Bestas & Sandra Wimberger & Dmitrii Degtev & Alexandra Madsen & Antje K. Rottner & Fredrik Karlsson & Sergey Naumenko & Megan Callahan & Julia Liz Touza & Margherita Francescatto & Carl Ivar Möl, 2023. "A Type II-B Cas9 nuclease with minimized off-targets and reduced chromosomal translocations in vivo," Nature Communications, Nature, vol. 14(1), pages 1-15, December.
    17. 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.

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