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GENE THERAPY

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.

References

  1. 1.

    Gillmore, J. D. et al. N. Engl. J. Med. https://doi.org/10.1056/NEJMoa2107454 (2021).

  2. 2.

    Doudna, J. A. & Charpentier, E. Science 346, 1258096 (2014).

    Article  Google Scholar 

  3. 3.

    Hirakawa, M. P., Krishnakumar, R., Timlin, J. A., Carney, J. P. & Butler, K. S. Biosci. Rep. 40, BSR20200127 (2020).

    CAS  Article  Google Scholar 

  4. 4.

    Frangoul, H. et al. N. Engl. J. Med. 384, 252–260 (2021).

    CAS  Article  Google Scholar 

  5. 5.

    Xu, L. et al. N. Engl. J. Med. 381, 1240–1247 (2019).

    CAS  Article  Google Scholar 

  6. 6.

    Stadtmauer, E. A. et al. Science. 367, eaba7365 (2020).

    CAS  Article  Google Scholar 

  7. 7.

    Cheng, Q. et al. Nat. Nanotechnol. 15, 313–320 (2020).

    CAS  Article  Google Scholar 

  8. 8.

    Dammes, N. et al. Nat. Nanotechnol. https://doi.org/10.1038/s41565-021-00928-x (2021).

  9. 9.

    Buning, H. & Srivastava, A. Mol. Ther. 12, 248–265 (2019).

    Google Scholar 

  10. 10.

    Buchholz, C. J., Friedel, T. & Buning, H. Trends Biotechnol. 33, 777–790 (2015).

    CAS  Article  Google Scholar 

  11. 11.

    Komor, A. C., Kim, Y. B., Packer, M. S., Zuris, J. A. & Liu, D. R. Nature 533, 420–424 (2016).

    CAS  Article  Google Scholar 

  12. 12.

    Anzalone, A. V. et al. Nature 576, 149–157 (2019).

    CAS  Article  Google Scholar 

Download references

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Correspondence to Hildegard Büning or Axel Schambach.

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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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