GBA1 and Gaucher disease type 1: Gaucher disease type 1, a common glycolipid storage disease, is caused by deleterious mutations in GBA1, which results in dysfunction of the lysosomal enzyme, glucocerebrosidase (GCase) and subsequent excess accumulation of glucosyl-ceramide (GluCer)/-sphingosine (GluSph) in various tissues [1].