Figure 2 | Scientific Reports

Figure 2

From: Splice modulating antisense oligonucleotides restore some acid-alpha-glucosidase activity in cells derived from patients with late-onset Pompe disease

Figure 2

Preliminary screening for the best PMO sequences that increased GAA activity in patientsderived fibroblasts. (a) The relative annealing positions of the AOs synthesised as PMOs are shown. PMOs are indicated as solid lines with the corresponding number shown above. Exons are shown as boxes and introns as lines (not drawn to scale). (b) The improvement of GAA activity in treated compared to untreated patient C 1 and C 2 derived fibroblasts. Data represent the mean fold change ±SDs of two biological replicates (n = 2). (c) qPCR, GAA enzyme activity (average fold change ±SDs of two biological replicates) and western analysis of patient C 1 and C 2 fibroblasts treated with different dosages of PMO 13, 15 and GTC (Gene Tools Control). UT; untreated. β tub; beta-tubulin, β act; beta-actin. The densitometric analysis of the western blot is shown as a bar graph below the Western image. The gels were cropped for presentation, and full-size gels are presented in Supplementary Fig. S5. GAA assays were performed in triplicate.

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