Mutation in the gene (GBA1) encoding for β-glucocerebrosidase (GCase), the lysosomal enzyme responsible for cleaving GluCer into glucose and ceramide (Figure 2C), leads to elevated levels of GluCer and is the underlying cause of the most common lysosomal storage disorder known as Gaucher’s disease (Vitner et al., 2010; Farfel-Becker et al., 2014). This evidence concerns the gene GBA1 and Gaucher disease.