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, Nancy D. Dalton, Anthony D. Rangel, Yunghang Chan, Jeremy Davis‐Turak, Jon C. Neumann, 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.
Scientific Reports · Jun 25, 2020Peer-reviewedMolecular Biology