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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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| Main Authors: | , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2006
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| Assuntos: | |
| Acesso em linha: | 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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