CRISPR-Cas9 generated Pompe knock-in murine model exhibits early-onset hypertrophic cardiomyopathy and skeletal muscle weakness.
Scientific Reports
Huang, Jeffrey Y JY; Kan, Shih-Hsin SH; Sandfeld, Emilie K EK; Dalton, Nancy D ND; Rangel, Anthony D AD; Chan, Yunghang Y; Davis-Turak, Jeremy J; Neumann, Jon J; Wang, Raymond Y RY