Figure 2: In vivo validation of ASOs targeting Dnm2 following intramuscular injection.
From: Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice
(a) Representative western blot of 7-week-old WT or Mtm1KO TA muscles injected with 20 μg of ASO control (ctrl), #1, #2 or #3. DNM2 is present as two bands in muscle tissue. DNM2 densitometries were quantified below and standardized to the loading control GAPDH (n=5–7 mice per group). (b) Photography of TA muscles from WT or Mtm1KO mice treated with ASO ctrl or ASO Dnm2 #1. (c) TA muscle weight relative to body weight (n=8). (d) Specific muscle force of the TA (n=5–6 mice per group).(e) TA muscle sections were stained with H&E (left) to visualize nuclei positioning or with SDH (right) for mitochondria oxidative activity distribution. Scale bars, 50 μm. (f) Percentage of fibres with mislocalized nuclei (n=4–6 mice per group). (g) Fibre area was determined in 600–1,000 fibres per sample (n=5 mice per group). Data represent means±s.e.m. NS, not statistically significant. *P<0.05, **P<0.01, ***P<0.001 for TA treated with ASO Dnm2 versus TA treated with ASO ctrl (ANOVA test). kDa, kilodalton; MW, molecular weight.
