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Therapeutic Benefit of Autophagy Modulation in Pompe Disease
The complexity of the pathogenic cascade in lysosomal storage disorders suggests that combination therapy will be needed to target various aspects of pathogenesis. The standard of care for Pompe disease (glycogen storage disease type II), a deficiency of lysosomal acid alpha glucosidase, is enzyme r...
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| Publicado no: | Mol Ther |
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| Main Authors: | , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Gene & Cell Therapy
2018
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6035739/ https://ncbi.nlm.nih.gov/pubmed/29804932 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.04.025 |
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