Data supporting Intravital imaging of muscle damage and response to therapy in a model of Pompe disease

Intravital microscopy provides insights into cellular processes in unperturbed tissues under physiological and pathological conditions. Here, we applied this powerful imaging tool for studying Pompe disease, a severe multisystem lysosomal glycogen storage disorder that is primarily marked by progressive deterioration of muscle tissues. We have used a reporter mouse model to directly visualize the limb muscle damage and response to therapy in anaesthetized live animals. Furthermore, we demonstrate that the disease progression and the treatment outcome can be monitored in vivo by noninvasive imaging of the clinically relevant tongue muscle. These results indicate that the reporter model provides a platform for efficient preclinical testing of different therapeutic interventions in Pompe disease.