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. 2021 Dec 10;50(1):17–34. doi: 10.1093/nar/gkab1199

Figure 3.

Figure 3.

Skipping efficacy and dystrophin rescue following 12-week repeated dosing of palm-tcDNA-ASO. (A) Quantification of exon 23 skipping levels in different muscle tissues 2-week after the end of the 12-week dosing regimen (palm-tcDNA-PO or palm-tcDNA-PS at 2, 4 or 10 μmol/kg/week) by RT-qPCR. Results are expressed as mean ± SEM; n = 4 mice/group. (B) ED50 values corresponding to the dose of ASO required to achieve 50% of exon 23 skipping in each tissue were determined with GraphPad Prism 7 software for palm-tcDNA-PO or palm-tcDNA-PS. (C) Detection and quantification of dystrophin restoration by western blot analysis in the different muscle tissues 2 weeks after the end of the 12-week dosing regimen (palm-tcDNA-PO or palm-tcDNA-PS at 2, 4 or 10 μmol/kg/week). The blot shows a representative example of dystrophin restoration in the quadriceps of one of the four animals per group. Results are expressed as mean ± SEM; n = 4 mice/group. (D) Detection of the dystrophin protein (green staining) by immunostaining on transverse sections of muscle tissues (quadriceps, diaphragm and heart) from WT and mdx mice treated with PBS or palm-tcDNA-PO or palm-tcDNA-PS for 12 weeks at 10 μmol/kg/week. Nuclei are labelled in blue (DAPI). Scale bar, 100 μm.