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Advancements in AAV-mediated Gene Therapy for Pompe Disease
Pompe disease (glycogen storage disease type II) is caused by mutations in acid α-glucosidase (GAA) resulting in lysosomal pathology and impairment of the muscular and cardio-pulmonary systems. Enzyme replacement therapy (ERT), the only approved therapy for Pompe disease, improves muscle function by...
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| Publicado no: | J Neuromuscul Dis |
|---|---|
| Main Authors: | , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
IOS Press
2020
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7029369/ https://ncbi.nlm.nih.gov/pubmed/31796685 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3233/JND-190426 |
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