Extended Data Fig. 2: Intramuscular injection of AAV-GFP has no effect on DM1 splicing events regulated by MBNL1. | Nature Biomedical Engineering

Extended Data Fig. 2: Intramuscular injection of AAV-GFP has no effect on DM1 splicing events regulated by MBNL1.

From: Reversal of RNA toxicity in myotonic dystrophy via a decoy RNA-binding protein with high affinity for expanded CUG repeats

Extended Data Fig. 2

(a) Representative Western blot and quantification of MBNL1 protein level in WT Gastrocnemius muscles five weeks after intramuscular injection of AAV9-GFP (1×1011 vg; n = 4) or saline. Data analyzed by unpaired Student’s t-test (ns: not significant). (b) Splicing profiles of Clcn1 exon 7a and Atp2a1 exon 22 assessed by RT-PCR in WT mice five weeks after intramuscular injection of AAV9-GFP (1×1011 vg) compared to saline vehicle-injected contralateral muscles or muscles form HSALR mice (n = 3-6). Data analyzed by one-way ANOVA followed by Tukey’s test (****p < 0.0001). (c) Modulation of 23 DM1-misspliced events regulated by MBNL1 in GA muscles of WT mice injected with AAV9-GFP-MBNL1∆ or AAV9-GFP compared to saline vehicle-injected contralateral muscles or muscles form HSALR mice (n = 3).

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