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Personalized medicine is having its day

From made-to-order genetic therapies to model organisms engineered to be ‘patient avatars’, the technology exists right now to save patients with rare diseases.

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Fig. 1: Workflow for N-of-1 therapeutic ASO development.

References

  1. Kim, J. et al. N. Engl. J. Med. 381, 1644–1652 (2019).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  2. Crooke, S. T. et al. Nat. Rev. Drug Discov. 20, 427–453 (2021).

    Article  CAS  PubMed  Google Scholar 

  3. Woodcock, J. & Marks, P. N. Engl. J. Med. 381, 1678–1680 (2019).

    Article  PubMed  Google Scholar 

  4. Brudvig, J. J. & Weimer, J. M. J. Clin. Invest. https://doi.org/10.1172/JCI157820 (2022).

    Article  PubMed  PubMed Central  Google Scholar 

  5. Korobeynikov, V. A., Lyashchenko, A. K., Blanco-Redondo, B., Jafar-Nejad, P. & Shneider, N. A. Nat. Med. 28, 104–116 (2022).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  6. Aartsma-Rus, A. et al. Nucleic Acid Ther. https://doi.org/10.1089/nat.2022.0060 (2022).

    Article  PubMed  PubMed Central  Google Scholar 

  7. Schor, N. F., Tamiz, A. P., Koroshetz, W. J., NINDS Ultra-Rare Gene-based Therapy (URGenT) Working Group & Broome, A.-M. Nat. Biotechnol. 39, 1497–1499 (2021).

    Article  CAS  PubMed  Google Scholar 

  8. Urnov, F. New York Times https://www.nytimes.com/2022/12/09/opinion/crispr-gene-editing-cures.html (9 December 2022).

Download references

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Seydel, C. Personalized medicine is having its day. Nat Biotechnol 41, 441–446 (2023). https://doi.org/10.1038/s41587-023-01724-9

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