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Adjunctive β2-agonists reverse neuromuscular involvement in murine Pompe disease
Pompe disease has resisted enzyme replacement therapy with acid α-glucosidase (GAA), which has been attributed to inefficient cation-independent mannose-6-phosphate receptor (CI-MPR) mediated uptake. We evaluated β2-agonist drugs, which increased CI-MPR expression in GAA knockout (KO) mice. Clenbute...
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Main Authors: | , , , , , , , |
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Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
Federation of American Societies for Experimental Biology
2013
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Assuntos: | |
Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3528321/ https://ncbi.nlm.nih.gov/pubmed/22993195 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1096/fj.12-207472 |
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