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. 2017 Jun 16;25(9):2117–2128. doi: 10.1016/j.ymthe.2017.05.019

Figure 5.

Figure 5

Reduction of Glycogen Accumulation in LO-GSDII Myotubes Carrying the c.-32-13T > G Mutation Treated with AMO 1+2+3

(A) GAA splicing profile assessed by RT-PCR in patient-derived myotubes treated with AMO 1+2+3 for 48 hr. WT myotubes were taken as positive control. The agarose picture is representative of two independent experiments. (B) qRT-PCR of the normal spliced (N) variant in LO-GSDII myotubes treated with AMO 1+2+3 for 48 hr. (C) GAA enzymatic activity in LO-GSDII myotubes treated with AMO 1+2+3 for 72 hr. (D) PAS staining of the LO-GSDII myotubes treated with AMO 1+2+3 for 7 days. The mock-treated LO-GSDII myotubes were taken as control. 10× magnification in the upper panels; 20× magnification in the lower panels. (D) Glycogen quantification in LO-GSDII myotubes treated with AMO 1+2+3 for 7 days using the fluorometric assay. The mock-treated LO-GSDII myotubes were taken as control. All data in (B), (C), and (E) are expressed as a percentage of the mock-treated LO-GSDII myotubes. In all experiments, the final combination AMO 1+2+3 was 15 μM. Data are represented in the graph as mean ± SEM of at least two independent experiments (*p < 0.05, **p < 0.01, ***p < 0.001).