The generation of induced pluripotent stem cells (iPSCs) from patients with infantile and late-onset types of Pompe disease and the effects of treatment with acid-α-glucosidase in Pompe's iPSCs
Keywords: PD; Pompe disease; GSDII; glycogen storage disease type II; GAA; acid-α-glycosidase; LSD; lysosomal storage disease; ERT; enzyme replacement therapy; iPSC; induced pluripotent stem cell; PAS; Periodic-acid-Schiff; ALP; alkaline phosphates; TEM; transmiss