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Salmeterol enhances the cardiac response to gene therapy in Pompe disease
Enzyme replacement therapy (ERT) with recombinant human (rh) acid α-glucosidase (GAA) has prolonged the survival of patients. However, the paucity of cation-independent mannose-6-phosphate receptor (CI-MPR) in skeletal muscle, where it is needed to take up rhGAA, correlated with a poor response to E...
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| Pubblicato in: | Mol Genet Metab |
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| Autori principali: | , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
2016
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4833676/ https://ncbi.nlm.nih.gov/pubmed/27017193 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2016.03.006 |
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