Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease

In Pompe disease, the deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA) causes skeletal and cardiac muscle weakness, respiratory failure, and premature death. While enzyme replacement therapy using recombinant human GAA (rhGAA) can significantly improve patient outcomes, detailed disea...

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Vydáno v:Commun Biol
Hlavní autoři: Wang, Jason, Zhou, Chris J., Khodabukus, Alastair, Tran, Sabrina, Han, Sang-Oh, Carlson, Aaron L., Madden, Lauran, Kishnani, Priya S., Koeberl, Dwight D., Bursac, Nenad
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group UK 2021
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Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC8100136/
https://ncbi.nlm.nih.gov/pubmed/33953320
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s42003-021-02059-4
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