Glycosphingolipids are endocytosed and targeted to the Golgi apparatus, but are
mistargeted to lysosomes in numerous sphingolipidoses. Substrate reduction therapy
utilizes imino sugars to inhibit glucosylceramide synthase and potentially abrogate the
effects of storage. Gaucher disease is a hereditary deficiency in glucocerebrosidase leading
to glucosylceramide accumulation; however, Gaucher fibroblasts exhibited normal Golgi
transport of lactosylceramide. To better understand the effects of glycosphingolipid …