Synergistic Efficacy from Gene Therapy with Coreceptor Blockade and a β(2)-Agonist in Murine Pompe Disease

Pompe disease (glycogen storage disease type II; acid maltase deficiency) is a devastating myopathy resulting from acid α-glucosidase (GAA) deficiency in striated and smooth muscle. Despite the availability of enzyme replacement therapy (ERT) with recombinant human GAA (rhGAA), the limitations of ER...

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Publicado en:Hum Gene Ther
Autores principales: Han, Sang-oh, Li, Songtao, Bird, Andrew, Koeberl, Dwight
Formato: Artigo
Lenguaje:Inglês
Publicado: Mary Ann Liebert, Inc. 2015
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Research Articles
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC4651047/
https://ncbi.nlm.nih.gov/pubmed/26417913
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2015.033
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