A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases - Kim-McManus, Olivia; Gleeson, Joseph G.; Mignon, Laurence; Smith Fine, Amena; Yan, Winston; Nolen, Nicole; Demarest, Scott; Berry-Kravis, Elizabeth; Finkel, Richard; Leonard, Stefanie; Finlayson, Samuel; Augustine, Erika; et al. (Nature Communications, 2024)
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