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

Huang JY, Kan SH, Sandfeld EK, Dalton ND, Rangel AD, Chan Y, Davis-Turak J, Neumann J, Wang RY
Sci Rep. 2020 Jun 25;10(1):10321. doi: 10.1038/s41598-020-65259-8. PubMed Article

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