Molecular BiologyScientific ReportsPeer-reviewed
CRISPR-Cas9 generated Pompe knock-in murine model exhibits early-onset hypertrophic cardiomyopathy and skeletal muscle weakness
Jeffrey Y. Huang, Shih‐hsin Kan, Emilie K. Sandfeld…·Raymond Wang
This paper describes a CRISPR-Cas9-generated murine model of infantile-onset Pompe Disease that exhibits early-onset hypertrophic cardiomyopathy and skeletal muscle weakness similar to the human condition.
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