Extended Data Fig. 3: Local and systemic administration of AAV-V5-MBNL1∆ corrects splicing defects in muscles of HSA^LR mice.

(a) Correction of Atp2a1 exon 22, Clcn1 exon 7a and Mbnl1 exon 5 alternative splicing assessed by RT-PCR in Gastrocnemius of HSA^LR mice after local intramuscular injection of AAV9-V5-MBNL1∆ (n = 3-4, upper panel) or AAV9-MBNL1∆ (n = 3, lower panel) and compared to saline vehicle-injected contralateral muscle or muscle from WT mice (n = 4). Data analyzed by one-way ANOVA followed by Tukey’s test (****p < 0.0001). (b) Correction of Atp2a1 exon 22 and Mbnl1 exon 5 alternative splicing misregulation in Gastrocnemius (GA) and Quadriceps (QUA) muscles of HSA^LR mice following systemic MBNL1∆ treatment (n = 5) compared to saline-injected HSA^LR (n = 4) and WT mice (n = 3). Data analyzed by one-way ANOVA followed by Tukey’s test (****p < 0.0001). (c) Levels of MBNL1 proteins assessed by Western blot in GA muscles of MBNL1∆-treated HSA^LR mice (n = 4) and saline-injected HSA^LR or WT mice (n = 3). Data analyzed by one-way ANOVA followed by Tukey’s test (ns: not significant).