Adeno-associated virus (AAV) mediated gene therapy continues to be a promising therapeutic path for various diseases, including monogenic neuromuscular and cardiac indications such as Duchenne muscular dystrophy (DMD) and Friedreich’s ataxia (FA). However, the high systemic doses currently required to achieve widespread therapeutic benefit can pose potential safety risks. These risks could be decreased or eliminated if therapeuticbenefit could be achieved using lower doses through a more targeted and efficacious vector.