Figure 1.

Dystrophin Restoration in mdx Mice with Yearlong Systemic Administration of PMO-GF

Dystrophin restoration in mdx mice treated with PMO-GF at the dosage of 50 mg/kg/week for 3 weeks followed by 50 mg/kg/month for 11 months intravenously. (A) Diagram to illustrate the dosing regimen and time for tissue collection. (B) Immunohistochemistry for dystrophin expression in body-wide muscles from mdx mice treated with PMO-GF (scale bar, 200 μm). (C) Representative western blot image to show dystrophin restoration in mdx mice treated with PMO-GF. 12.5, 25, and 50 μg of total protein from C57BL6 and 50 μg from untreated and treated mdx muscle samples were loaded, respectively. α-Actinin was used as the loading control. (D) Quantitative analysis of western blot results with ImageJ. Four biological replicates were examined and quantified based on densitometry as described in the Materials and Methods. The data present as mean + SEM unless otherwise specified (n = 4). (E) Quantification of PMO in muscle tissues with ELISA. No significant difference was detected between 6-month and yearlong treatment of PMO-GF (n = 4). A, abdominal muscle; D, diaphragm; Q, quadriceps; G, gastrocnemius; i.v., intravenous injection; T, triceps; TA, tibialis anterior.