The first preliminary evidence that in vivo gene editing in a clinical setting is feasible with no early signs of severe adverse events comes from an ongoing clinical trial to treat transthyretin amyloidosis, a fatal monogenetic disease.
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Büning, H., Schambach, A. A first step toward in vivo gene editing in patients. Nat Med 27, 1515–1517 (2021). https://doi.org/10.1038/s41591-021-01476-6
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DOI: https://doi.org/10.1038/s41591-021-01476-6