A beta-blocker, propranolol, decreases the efficacy from enzyme replacement therapy in Pompe disease

Enzyme replacement therapy (ERT) with recombinant human acid α-glucosidase (rhGAA) fails to completely reverse muscle weakness in Pompe disease. β2-agonists enhanced ERT by increasing receptor-mediated uptake of rhGAA in skeletal muscles. To test the hypothesis that a β-blocker might reduce the effi...

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Veröffentlicht in:Molecular genetics and metabolism 2016-02, Vol.117 (2), p.114-119
Hauptverfasser: Han, Sang-oh, Pope, Rand, Li, Songtao, Kishnani, Priya S., Steet, Richard, Koeberl, Dwight D.
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Sprache:eng
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Zusammenfassung:Enzyme replacement therapy (ERT) with recombinant human acid α-glucosidase (rhGAA) fails to completely reverse muscle weakness in Pompe disease. β2-agonists enhanced ERT by increasing receptor-mediated uptake of rhGAA in skeletal muscles. To test the hypothesis that a β-blocker might reduce the efficacy of ERT, because the action of β-blockers opposes those of β2-agonists. Mice with Pompe disease were treated with propranolol (a β-blocker) or clenbuterol in combination with ERT, or with ERT alone. Propranolol-treated mice had decreased weight gain (p
ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2015.09.012