Figure 1.

Restoration of Functional Glycosylation of α-DG in the P448Lneo⁻ Mouse after AAV9-mhFKRP Treatment

(A) Immunohistochemistry with antibodies (FKRP-Stem and IIH6) to FKRP and F-α-DG demonstrating dose-dependent levels of FKRP and F-α-DG signals in the majority of muscle fibers of the heart, diaphragm, and quadriceps treated with 1e13 and 5e13 vg/kg. Less than 1% of fibers with strong signal across section areas were detected in skeletal and cardiac muscles from mice treated with the lower doses of AAV9. The majority of the fibers in the diaphragm treated with both lower and higher doses of AAV9 lacks clear mhFKRP detection yet shows strong IIH6 detection. Untreated P448Lneo⁻ mice were used as controls (top left panel). The scale bars represent 200 μm. (B) Variable and dose-dependent levels of mhFKRP and F-α-DG expression were confirmed by western blot. A clear pattern of α-DG glycosylation can be observed in each tissue by both doses as compared to the untreated control to levels near that of the WT control (C57). TA, tibialis anterior muscle.