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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 no: | Hum Gene Ther |
|---|---|
| Main Authors: | , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Mary Ann Liebert, Inc.
2015
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| Assuntos: | |
| Acesso em linha: | 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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