… The only available therapy for Pompe disease is enzyme replacement therapy (… autophagic
gene, Atg7, is inactivated in skeletal muscle. Suppression of autophagy in skeletal muscle of …

… autophagy—have become the foundation of our understanding of … of large pools of autophagic
debris in skeletal muscle and the … of using recombinant enzyme to treat skeletal muscle. …

… enzyme replacement therapy (ERT) for Pompe disease as well as other LSDs since the
recombinant enzymes … Our studies pointed to the role of glycogen autophagy in skeletal muscle …

… autophagic process and leading to accumulation of undegraded products [10,36]. In skeletal
muscle … also present in autophagic vacuoles containing cytoplasmic degradation products. …

… Currently, the only approved treatment for Pompe disease is enzyme replacement therapy
(ERT… Autophagy and mistargeting of therapeutic enzyme in skeletal muscle in Pompe disease. …

… patients is enzyme replacement therapy (ERT) which aims to … of autophagy to the
pathophysiology of Pompe disease. … the regenerative capacity of skeletal muscle in Pompe …

… -enzyme fusion proteins as a means to target the enzyme to … To examine autophagy in skeletal
muscle, western blotting … cardiac and skeletal muscle, ie the target tissues for PD therapy. …

… may yield novel therapeutic targets for these disorders. … ubiquitin ligase like conjugating
enzyme autophagy-related 10 (… defective autophagic pathway in patients with Pompe Disease …

… in treating skeletal muscle in Pompe disease are now well recognized, and new therapeutic
… reversal of the autophagic defect in the muscles affected by Pompe disease remains to be …

… enzyme replacement therapy (ERT) in Pompe disease (PD); however, its efficacy is limited
in skeletal muscle because of a partial blockage of autophagic … enzyme delivery. Adjunctive …