CRISPR-mediated generation and characterization of a Gaa homozygous c.1935C>A (p.D645E) Pompe disease knock-in mouse model recapitulating human infantile onset-Pompe disease. - Kan SH; Huang JY; Harb J; Rha A; Dalton ND; Christensen C; Chan Y; Davis-Turak J; Neumann J; Wang RY (Scientific reports, 2022)