Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice.
Elmallah MK, Falk DJ, Nayak S, Federico RA, Sandhu MS, Poirier A, Byrne BJ, Fuller DD. Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice. Mol Ther. 2014 Apr; 22(4):702-12.