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A first step toward in vivo gene editing in patients

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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Fig. 1: In vivo and ex vivo strategies for CRISPR–Cas9-based gene editing.


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Correspondence to Hildegard Büning or Axel Schambach.

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The authors declare no competing interests.

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Büning, H., Schambach, A. A first step toward in vivo gene editing in patients. Nat Med 27, 1515–1517 (2021).

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