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.
Kim-McManus O, et al. Among authors: yan w.
Nat Commun. 2024 Nov 12;15(1):9802. doi: 10.1038/s41467-024-54077-5.
Nat Commun. 2024.
PMID: 39532857
Review.