Oligonucleotides offer therapeutic potential for patients with genetic disorders carrying unique mutations, but developing individualized therapies is not supported by the current process for drug development.
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I thank R. Collin, Y. Elgersma, H. Graessner, W. van Roon-Mom, R. Schüle, M. Synofzik and T. Yu for useful discussions.
A.A.-R. has disclosures related to patents, consultancies, advisory-board memberships and honoraria. See http://www.exonskipping.nl/disclosures for details.
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Aartsma-Rus, A. ‘N of 1’ therapies need a better model. Nat Med 27, 939 (2021). https://doi.org/10.1038/s41591-021-01380-z