Figure 1.

AT-GAA Cleared Glycogen Accumulation and Restored Normal Levels of Endosomal/Lysosomal Membrane Markers in Muscle from KO Mice

Muscle biopsies were collected from age and sex-matched WT, untreated (KO), and AT-GAA-treated KO (KO-ERT) mice. (A) Glycogen content in muscles from WT, untreated KO (KO), and treated KO (KO-ERT) mice (n = 6 for each group). (B) Muscle strength was assessed using grip-strength test after 8 and 9 administrations of the compound (n = 5 WT; n = 4 KO; n = 7 KO-ERT). (C) GAA activity in muscle tissues. (D) Western blot analysis of muscle lysates from WT, untreated KO (KO), and treated KO (KO-ERT) mice (n = 6 for each group) with the indicated antibodies. GAPDH was used as loading control. Each data point represents an individual mouse. Statistical significance was determined by one-way ANOVA. Graphs represent mean ± SD. ∗p < 0.05; ∗∗p < 0.01; ∗∗∗p < 0.001; ∗∗∗∗p < 0.0001.