To verify the antisense efficacy on mRNA expression, we carried out RT-qPCR quantification for DUX4 and two downstream genes that have been previously demonstrated to be activated in FSHD animal models, Trim43 and Wfdc3 (Fig. 4A–C) (3,32,42). This evidence concerns the gene DUX4 and facioscapulohumeral muscular dystrophy.