Fig. 1: FDC reduces WT Dmpk expression in mouse skeletal muscle to a greater extent than ADC and enhances ASO delivery via a TfR1-mediated mechanism. | Communications Medicine

Fig. 1: FDC reduces WT Dmpk expression in mouse skeletal muscle to a greater extent than ADC and enhances ASO delivery via a TfR1-mediated mechanism.

From: FORCE platform overcomes barriers of oligonucleotide delivery to muscle and corrects myotonic dystrophy features in preclinical models

Fig. 1

WT male mice were administered by tail IV injection on day 0 with 10 mg/kg ASO-equivalents of a FDC4 (blue bars), ADC1 (green bars) or vehicle (gray bars), or b ASO1 (red bars), FDC5 (teal bars), and FDC4 (blue bars), and sacrificed on day 14 for analysis of gene expression. Total RNA extracted from the indicated muscles was reverse-transcribed and Dmpk RNA expression was measured by qPCR using Ppib as the reference gene. Expression in each muscle from mice injected with FDC4 or ADC1 was normalized to Dmpk expression in the corresponding muscle of mice administered with vehicle. Data are means + SD; data were analyzed by one-way ANOVA followed by uncorrected Fisher’s LSD test, statistically significant differences from vehicle are indicated with exact P values. Each circle represents an individual mouse.

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