Genetic Suppression of Autophagy Dramatically Enhances Enzyme Replacement Therapy in Murine Pompe Disease

摘要

Classic autophagy or macroautophagy is an evolutionarily conserved major intracellular system by which a portion of cytoplasm and damaged organelles are delivered to lysosomes for degradation and recycling. Lately, autophagy has attracted much attention due to its role in a variety of physiological processes and its connection with a range of human diseases. Our recent data provide strong evidence for the role of autophagy in the pathogenesis of Pompe disease, a lysosomal glycogen storage disorder caused by deficiency of acid a-glucosidase (GAA).