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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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| Publicado no: | Commun Biol |
|---|---|
| Main Authors: | , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Nature Publishing Group UK
2021
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| Assuntos: | |
| Acesso em linha: | 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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