Figure 1.

Study Design and Rescue of α-DG Glycosylation in Dystrophic Mice

(A) Experimental design of FKRP^P448L mutant mice injected with AAV9-FKRP at 5 (T18, n = 4), 13 (T26, n = 4), 26 (T39, n = 4), and 39 weeks of age (T52, n = 4) in a 13-week treatment study. (B) Immunofluorescence staining of glycosylated α-DG in tibialis anterior, diaphragm, and heart tissue from indicated mice. Scale bars, 200 μm. (C) Western blot analysis of glycosylated α-DG expression in tibialis anterior, diaphragm, and heart tissue from FKRP^P448L mutant mice injected with AAV9-FKRP (+) or untreated (−). Glycosylated α-DG was detected with the IIH6C4 antibody at a dilution of 1:200 and 1:1,000 for immunofluorescence staining and western blot, respectively. Anti-actin antibody was used as the protein loading control. (D) Western blot analysis of exogenous FKRP protein in tibialis anterior tissues from indicated mice. FKRP was detected with an FKRP (C-terminal) antibody at a dilution of 1:400. Anti-GAPDH antibody was used as the protein loading control. (E) FKRP transgene expression in tibialis anterior tissues was analyzed by quantitative real-time PCR methods. All levels are relative to those in untreated FKRP^P448L mutant mice cohorts and have been normalized using GAPDH.