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Personalized medicine is having its day

Nature Biotechnology volume 41, pages 441–446 (2023)Cite this article

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.

Last October, Susannah Rosen, an 8-year-old girl with a devastating neurodegenerative genetic disease, took her first dose of a custom-made genetic medicine. What’s more, she got it at no cost, provided by the non-profit foundation n-Lorem. The drug isn’t gene therapy: it doesn’t change Susannah’s genome or undo her mutation. It’s an antisense oligonucleotide (ASO), a short sequence of modified DNA, designed to bind to a problematic mRNA transcript and stop production of the disease-causing protein.

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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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  1. Los Angeles, CA, USA

    Caroline Seydel

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  1. Caroline Seydel
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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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