Base and prime editors extend the therapeutic scope of genome editing. Experience from CRISPR clinical trials offers a roadmap for their translation, from manufacturing and quality control to clinical trial design and regulatory assessment.
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References
Locatelli, F. et al. Exagamglogene autotemcel for transfusion-dependent β-thalassemia. N. Engl. J. Med. 390, 1663–1676 (2024).
Frangoul, H. et al. Exagamglogene autotemcel for severe sickle cell disease. N. Engl. J. Med. 390, 1649–1662 (2024).
Frangoul, H. et al. CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia. N. Engl. J. Med. 384, 252–260 (2021).
Cohn, D. M. et al. CRISPR-based therapy for hereditary angioedema. N. Engl. J. Med. 392, 458–467 (2025).
Pierce, E. A. et al. Gene editing for CEP290-associated retinal degeneration. N. Engl. J. Med. 390, 1972–1984 (2024).
Pacesa, M., Pelea, O. & Jinek, M. Past, present, and future of CRISPR genome editing technologies. Cell 187, 1076–1100 (2024).
Abou-el-Enein, M. The fate(s) of CAR T-cell cherapy: navigating the risks of CAR+ T-cell malignancy. Blood Cancer Discov. 5, 249–257 (2024).
Musunuru, K. et al. Patient-specific in vivo gene editing to treat a rare genetic disease. N. Engl. J. Med. 392, 2235–2243 (2025).
Cadinanos-Garai, A. et al. High-dimensional temporal mapping of CAR T cells reveals phenotypic and functional remodeling during manufacturing. Mol. Ther. 33, 2291–2309 (2025).
Mackall, C. L. et al. Enhancing pediatric access to cell and gene therapies. Nat. Med. 30, 1836–1846 (2024).
Acknowledgements
M.A. is an inventor on patents unrelated to this work. M.A. is supported in part by the National Cancer Institute under award no. P30CA014089. The content is solely the responsibility of the authors and does not necessarily reflect the official views of the National Cancer Institute or the National Institutes of Health.
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Flugel, C.L., Cadinanos-Garai, A. & Abou-el-Enein, M. A clinical roadmap for base and prime editing. Nat Rev Bioeng 3, 914–916 (2025). https://doi.org/10.1038/s44222-025-00360-z
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DOI: https://doi.org/10.1038/s44222-025-00360-z
