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. 2022 Jan 21;50(10):5443–5466. doi: 10.1093/nar/gkac018

Figure 6.

Figure 6.

PN backbone chemistry enhances muscle exposure. (A) Concentration of the indicated oligonucleotide detected in gastrocnemius, diaphragm, and heart muscle in mdx-2 study. Dots represent individual mice; box and whiskers show min to max. Mean ± s.d. n ≥ 4. Stats performed with pairwise comparisons to PBS. (B) Relationship between tissue exposure and exon-skipping activity for the indicated oligonucleotides in gastrocnemius, diaphragm, and heart muscle in mdx-2 study. (C) Images showing DMD-3034 and DMD-2788 in gastrocnemius, diaphragm, and cardiac muscle after ViewRNA staining from mdx-1 study at day 7. Tissues treated with PBS are also shown. Arrows demarcate myoblast nuclei that are positive for DMD-2788. Scale bars are 20 μm. (D) Concentration of DMD-3034 or DMD-2788 detected in whole cell (left), nuclear (middle), or cytoplasmic (right) extracts from differentiated myoblasts treated for 24 h as quantified by hybridization ELISA. n = 3 mean ± s.e.m. Unpaired, two-tailed t test *P < 0.05, **P < 0.01, *** P < 0.001. (E) Concentration of DMD-3034 or DMD-2788 in whole cell extracts from myoblasts treated for 3 h and washed for the indicated time. n = 3 mean ± s.e.m. * P < 0.05, ** P < 0.01, **** P < 0.0001 Two-way ANOVA with multiple comparisons.