… therapies to treat heart disease. The lack of knowledge in the molecular mechanisms of the
disease … Autophagy is a highly conserved homeostatic process that plays an essential role in …

… diseases, and autophagy plays a crucial role in maintaining typical cardiac structure, function,
and therapy … A total of 803 autophagy-related genes were obtained as the autophagy gene …

… (GAA) deficiency (Pompe disease), which is characterized by … AP-3-deficient cells show
mistargeting of some lysosomal … dependent on autophagy and essential autophagy proteins like …

… described the updated diagnosis and therapy on ML II/III. … A specific disease caused by a
defective uncovering enzyme … Niemann–Pick C2 protein maintains lysosomal and autophagic …

… of autophagic material and glycogen in skeletal and cardiac … While the latter is elevated in
Pompe disease, this biomarker … to the commencement of enzyme replacement therapy (ERT). …

… We will not discuss the role of lysosomes in autophagy … (UPR mt ), activated by the mistargeting
of proteins (UPR am ) … ), a lysosomal enzyme mutated in Pompe's disease whose loss …

… on inherited diseases and for testing therapeutic agents. … of late-onset Pompe disease, which
is characterised by slow … did not fully exhibit the autophagic abnormalities that are observed …

… of autophagy and plays an important role in induced autophagy. … , caused by a deficiency of
glycogenic glycemic enzyme acid … as a therapeutic target to modulate autophagy in diseases …

… impeded the efficacy of Pompe disease gene therapy. Long-… skeletal muscle, which correlated
with improved autophagy … due to increased risk of fatal mistargeting. To circumvent this …

… impeded the efficacy of Pompe disease gene therapy. Long-… skeletal muscle, which correlated
with improved autophagy … due to increased risk of fatal mistargeting. To circumvent this …