GBA1 and lysosomal storage disease: Gaucher disease (GD) is an autosomal recessive lysosomal storage disorder caused by mutations in the acid β-glucosidase gene (GBA1), resulting in the deficient activity of acid β-glucosidase (GCase), and the subsequent progressive accumulation of glucosylceramide (GlcCer) and glucosylsphingosine (LysoGL1) within the lysosomes, mainly in cells of the reticuloendothelial lineage [1,2].