Figure 4.

Assessment of Cardiac and Motor Functions in GAA^−/− Mice Treated with LV.LCR-EFS.GAA HSC Gene Therapy

(A) Representative M-mode images showing septum, left ventricular space, and left ventricular posterior wall (LVPW) of wild-type, GAA^−/−, and LV.LCR-EFS.GAA (LCR)-treated mice during diastole (d) and systole (s). (B–D) Heart mass and percentage of heart mass on total body weight (B), left ventricle (LV) mass (C), and thickness (D) of the LVPW of LCR-treated mice compared to wild-type and GAA^−/− littermates. (E and F) Motor coordination assessed by rotarod (E) and muscle strength (F) measured by grip strength meter of LCR-treated mice compared to wild-type and GAA^−/− littermates. Cardiac performances were evaluated via echocardiography in mice that showed VCN > 0.8 (details in Table S1). Data are shown as means ± SEM of three independent experiments with number of mice (n =) indicated in the graphs.