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. 2010 Aug 10;18(10):1822–1829. doi: 10.1038/mt.2010.151

Figure 1.

Figure 1

Systemic administration of B-MSP-PMO conjugate at single and multiple 3 mg/kg doses in mdx mice. Dystrophin expression following single and six biweekly multiple 3 mg/kg injections of the B-MSP-PMO conjugate in adult mdx mice. (a) Immunostaining of muscle tissue cross-sections to detect dystrophin protein expression and localization in mdx mice treated with single injection (the third panel, single) and multiple injections of B-MSP-PMO conjugates (bottom panel, multiple). Data from control normal C57BL6 and untreated mdx mice were shown (first and the second panel, respectively). Muscle tissues analyzed were from tibialis anterior (TA), quadriceps, triceps, biceps, abdominal wall (abdominal), and diaphragm and heart muscles. Bar = 200 µm. (b) Percent of dystrophin-positive fibers in analyzed muscle tissue cross-sections from mdx mice treated with single and multiple 3 mg/kg B-MSP-PMO conjugate doses (six sections per tissue/muscle analyzed). (c) RT-PCR analysis to detect dystrophin exon-skipping transcripts in the treated tissues with B-MSP-PMO. Unskipped and skipped transcript products as indicated (exon-skipped bands indicated by Δexon23—for exon 23 deleted; Δexon22 + 23—for exons 22 and 23 deleted). (d) Western blot for detecting the level of dystrophin protein restored in the analyzed tissues from mdx mice treated with multiple 3 mg/kg B-MSP-PMO conjugate doses compared with C57BL6 and untreated mdx control mice. 50 µg protein was loaded for each sample except for C57BL6 control lane where 2.5 µg of protein was loaded. α-Actinin was used as a loading control (six mice in each group). MSP, muscle-specific heptapeptide; PMO, phosphorodiamidate morpholino; RT, reverse transcriptase.