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GENE THERAPY

A first step toward in vivo gene editing in patients

Nature Medicine volume 27, pages 1515–1517 (2021)Cite this article

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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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Author information

Author notes

  1. These authors contributed equally: Hildegard Büning, Axel Schambach.

Authors and Affiliations

  1. Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany

    Hildegard Büning & Axel Schambach

  2. REBIRTH Research Center for Translational Regenerative Medicine, Hannover Medical School, Hannover, Germany

    Hildegard Büning & Axel Schambach

  3. Division of Hematology/Oncology, Boston Children’s Hospital, Harvard Medical School, Boston, MA, USA

    Axel Schambach

Authors
  1. Hildegard Büning
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  2. Axel Schambach
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Corresponding authors

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). https://doi.org/10.1038/s41591-021-01476-6

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