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. 2024 Aug 28;67(17):14868–14884. doi: 10.1021/acs.jmedchem.4c00803

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© 2024 The Authors. Published by American Chemical Society

Permits non-commercial access and re-use, provided that author attribution and integrity are maintained; but does not permit creation of adaptations or other derivative works (https://creativecommons.org/licenses/by-nc-nd/4.0/).

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Impact of antibody conjugation site on PMO exon-skipping efficacy. (a) 3′ conjugation. (b) 5′ conjugation. (c) Impact of conjugate site on skipping efficiency in gastrocnemius. (d) 3′ PEG2-maleimide conjugation. (e) Impact of conjugation site on gastrocnemius tissue concentration. For (c) and (e), n = 4 per group, error bars represent SEM. DIPEA, N,N-diisopropylethylamine; DMSO, dimethyl sulfoxide; MCC, 4-(N-maleimidomethyl) cyclohexane-1-carboxylate; PEG2, polyethylene glycol 2-maleimide linker; PBS, phosphate buffered saline; SCRAM, scramble PMO control; SEM, standard error of the mean; RT, room temperature.