Fig. 2: Skeletal muscle improvement after DG9-PMO treatment.
From: DG9 boosts PMO nuclear uptake and exon skipping to restore dystrophic muscle and cardiac function
a Eight week-old male hDMDdel45;mdx mice were treated retro-orbitally four times once/week with either saline, DG9-PMO, or PMO at 30 mg/kg bw. Saline-treated WT served as control. Functional tests were conducted before and 1 week after treatment. Blood and urine samples were collected 10 days post-treatment. Tissues were collected 2 weeks after the last injection. All mice received a 15 mg/kg subcutaneous injection of β-isoproterenol and 1% Evans blue dye intraperitoneally 24 h before sacrifice. b Forelimb and total grip strength of individual groups of mice normalized to their body weight after treatment. Changes in endurance and coordination compared to baseline measured using treadmill and rotarod (n = 4–6). c Quantification of exon 44 skipping by RT-PCR (left) and dystrophin restoration by western blot (right) in various skeletal muscles after treatment (n = 5). Dystrophin (Leica, NCL-DYS1) restoration presented relative to wild-type control after normalization with desmin (Abcam, ab8592). Statistical analysis: One-way ANOVA with Tukey’s test. Not significant, P > 0.05 (not shown). Error: SEM (b, c). d Representative immunofluorescence images for dystrophin (ab15277) in TA muscles. Percentage of dystrophin-positive fibers calculated in relation to laminin. Scale 200 µm. Source data are provided as a Source Data file.
