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Differences in the predominance of lysosomal and autophagic pathologies between infants and adults with Pompe disease: implications for therapy
Pompe disease is a lysosomal storage disorder caused by the deficiency of acid alpha-glucosidase, the enzyme that degrades glycogen in the lysosomes. The disease manifests as a fatal cardiomyopathy and skeletal muscle myopathy in infants; in milder late-onset forms skeletal muscle is the major tissu...
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| Main Authors: | , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2010
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2991562/ https://ncbi.nlm.nih.gov/pubmed/20801068 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2010.08.001 |
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