Evaluation of muscle satellite cell targeting
Correcting genetic defects within the endogenous muscle SCs could provide a long-term therapy for muscle disorders with potential to repair a patient’s muscles over his/her lifetime. Although a number of studies have now shown muscle SC targeting is feasible using AAV, the efficiency is low, and it is still not known if these studies efficiently targeted quiescent muscle SCs and if they remain in the niche and maintain the ability to continuously restore dystrophin after multiple rounds of injury. We are exploring approaches to improve SC targeting in vivo.