Correction of the enzymatic and functional deficits in a model of pompe disease using adeno-associated virus vectors

Thomas J. Fraites, Mary R. Schleissing, R. Andrew Shanely, Glenn A. Walter, Denise A. Cloutier, Irene Zolotukhin, Daniel F. Pauly, Nina Raben, Paul H. Plotz, Scott K. Powers, Paul D. Kessler, Barry J. Byrne