Fig. 4. Enhanced efficacy from ERT plus clenbuterol treatment.
Male GAA-KO mice were administered four weekly doses of 20 mg/kg body weight of rhGAA and sacrificed one week following the last injection. Groups of mice were treated with clenbuterol (n=6) or untreated (n=5) during ERT. Mice were euthanized for tissue analysis 4 weeks after vector injection. (A) GAA enzyme levels and (B) glycogen content were evaluated in the target tissues, including the tibialis anterior (Tib. Ant.). Statistically significant alterations associated with clenbuterol treatment indicated (*).