CRISPR-Cas9 generated Pompe knock-in murine model exhibits early-onset hypertrophic cardiomyopathy and skeletal muscle weakness

Infantile-onset Pompe Disease (IOPD), caused by mutations in lysosomal acid alpha-glucosidase (Gaa), manifests rapidly progressive fatal cardiac and skeletal myopathy incompletely attenuated by synthetic GAA intravenous infusions. The currently available murine model does not fully simulate human IO...

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Detalhes bibliográficos
Publicado no:Sci Rep
Main Authors: Huang, Jeffrey Y., Kan, Shih-Hsin, Sandfeld, Emilie K., Dalton, Nancy D., Rangel, Anthony D., Chan, Yunghang, Davis-Turak, Jeremy, Neumann, Jon, Wang, Raymond Y.
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group UK 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7316971/
https://ncbi.nlm.nih.gov/pubmed/32587263
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-020-65259-8
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