Autophagy and Mis-targeting of Therapeutic Enzyme in Skeletal Muscle in Pompe Disease

Enzyme replacement therapy (ERT) became a reality for patients with Pompe disease, a fatal cardiomyopathy and skeletal muscle myopathy caused by a deficiency of glycogen-degrading lysosomal enzyme acid alpha-glucosidase (GAA). The therapy, which relies on receptor-mediated endocytosis of recombinant...

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Autores principales: Fukuda, Tokiko, Ahearn, Meghan, Roberts, Ashley, Mattaliano, Robert J., Zaal, Kristien, Ralston, Evelyn, Plotz, Paul H., Raben, Nina
Formato: Artigo
Lenguaje:Inglês
Publicado: 2006
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC2693339/
https://ncbi.nlm.nih.gov/pubmed/17008131
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2006.08.009
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