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. 2019 Mar 3;6(4):642–654. doi: 10.1002/acn3.738

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© 2019 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals, Inc on behalf of American Neurological Association.

This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.

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Antisense oligonucleotide‐mediated skipping of PE50.1 in patient iFDM cells. (A) Pre‐mRNA DYSF transcript and AON1, AON2 and AON3 (Table S2), which target potential exonic splicing enhancers in PE50.1 as shown. Primers in exon 50 (16F) and 51(15R) amplify cDNAs to distinguish normal mRNA transcripts (88 bp product containing exon 50 + 51) from mutant PE50.1 transcripts (268 bp product containing exon 50 + PE50.1 + 51). (B) RT‐PCR analysis of mRNA splicing. Patient JF196 iFDM cells treated with AON1, AON2 and AON3 (600 nmol/L, duplicate cultures for each) expressed reduced amounts of PE50.1 mutant mRNA and higher normal DYSF mRNA compared with a non‐specific scrambled (SCR, 600 nmol/L) AON‐treated, TE‐treated, or no treatment cells. Normal (NHDF‐2) iFDMs only expressed normal DYSF transcripts. iFDMs were allowed to differentiate in DM for 6 days then treated with AONs in DM for an additional 2 days. (‐RT), no reverse transcriptase; H₂O, no RNA used in RT reactions.