Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice

Pompe disease is an autosomal recessive genetic disorder characterized by a deficiency of the enzyme responsible for degradation of lysosomal glycogen (acid α-glucosidase (GAA)). Cardiac dysfunction and respiratory muscle weakness are primary features of this disorder. To attenuate the progressive a...

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Bibliographic Details
Published in:Mol Ther Methods Clin Dev
Main Authors: Falk, Darin J, Soustek, Meghan S, Todd, Adrian Gary, Mah, Cathryn S, Cloutier, Denise A, Kelley, Jeffry S, Clement, Nathalie, Fuller, David D, Byrne, Barry J
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2015
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4445006/
https://ncbi.nlm.nih.gov/pubmed/26029718
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2015.7
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