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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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Detalhes bibliográficos
Main Authors: Li, Songtao, Sun, Baodong, Nilsson, Mats I., Bird, Andrew, Tarnopolsky, Mark A., Thurberg, Beth L., Bali, Deeksha, Koeberl, Dwight D.
Formato: Artigo
Idioma:Inglês
Publicado em: Federation of American Societies for Experimental Biology 2013
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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