GAA and Glycogen storage disease due to acid maltase deficiency: Pompe disease has been predominantly modeled in the GAA knockout mice (Raben et al., 2001) that recapitulate key disease features such as striated muscle and nervous tissue glycogen accumulation (DeRuisseau et al., 2009), lysosomal abnormalities (Doyle et al., 2019), neuropathology (DeRuisseau et al., 2009), cardiac defects (Han et al., 2016), and muscle weakness (Lee et al., 2018).