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A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases.

Kim-McManus O, Gleeson JG, Mignon L, Smith Fine A, Yan W, Nolen N, Demarest S, Berry-Kravis E, Finkel R, Leonard S, Finlayson S, Augustine E, Lyon GJ, Schule R, Yu T. A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases. Nat Commun. 2024 11 12; 15(1):9802.

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