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N of 1’ therapies need a better model

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.

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Correspondence to Annemieke Aartsma-Rus.

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A.A.-R. has disclosures related to patents, consultancies, advisory-board memberships and honoraria. See for details.

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Aartsma-Rus, A. ‘N of 1’ therapies need a better model. Nat Med 27, 939 (2021).

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