COMPOSITIONS AND METHODS FOR TREATING GLYCOGEN STORAGE DISEASES

摘要

Compositions and methods of use include therapeutically effective molecules for treatment of diseases in mammals including glycogen storage diseases (e.g., Pompe disease). These compositions in combination with various routes and methods of administration result in targeted uptake and expression of therapeutic molecules in specific organs, tissues and cells resulting in correction of a disease such as a glycogen storage disease (e.g., Pompe disease).