Figure 4.

H&E Staining and Immunohistochemical Detection of F-α-DG and mhFKRP Expression in C57BL/6 (WT) and P448Lneo⁻ Mice Six Months after AAV9-mhFKRP Treatment

(A) H&E staining, left panel. Detection of mhFKRP (red) with FKRP-Stem antibody and F-α-DG (green) with IIH6 antibody in untreated, 1e13 vg/kg, or 5e13 vg/kg treated quadriceps, diaphragm, and heart tissues is shown. Dose-dependent restoration of F-α-DG was observed in P448Lneo⁻ tissues. Mutant hFKRP was not clearly detected in the treated WT mice, and no change was observed in the IIH6 signal between the treated and control WT mice. The scale bars represent 100 μm. (B) Detection of mhFKRP and F-α-DG in quadriceps, diaphragm, and heart by western blot is shown. Enhanced F-α-DG was detected in the mhFKRP-treated P448Lneo⁻ mice. Mutant hFKRP was detected only in the heart of higher dose AAV9-mhFKRP-treated WT mice.