The data we present here demonstrate that P2RX7 ablation in the most widely used animal model of DMD produced significant improvements in key functional and molecular disease parameters in dystrophic leg muscles at 4 wk and in legs, diaphragms, and hearts at 20 mo, i.e., at all the stages where the model reproduces the DMD pathology [21]. The gene discussed is P2RX7; the disease is Duchenne muscular dystrophy.