Extended Data Fig. 4: Analysis of DM1 splicing events in heart, liver and kidney of HSA^LR mice treated systemically with AAV-MBNL1∆.

(a) Splicing profiles of Scn5a exon 6b, Mbnl1 exon 5, Dmd exon 78 and Mbnl2 exon 5 in heart following systemic injection of AAV-V5-MBNLΔ or saline vehicle in HSA^LR mice (n = 5-6) and compared to WT mice injected with saline vehicle (n = 3) (b) Splicing profiles of Mbnl1 exon 5 and Mbnl2 exon 5 in kidney (K) and liver (L) assessed by RT-PCR following systemic injection of AAV-V5-MBNLΔ or saline vehicle in HSA^LR mice (n = 3-4), and compared to WT mice injected with saline vehicle (n = 2-3). Data analyzed by one-way ANOVA followed by Tukey’s test (ns = not significant).