N-ButyldeoxynojirimycinHCl is a novel inhibitor of UGCG (glucosylceramide synthase) as well as Glucosidase I and II (α-glucosidase I and II). The compound has been shown to inhibit glucosylceramide synthase without ceramide accumulation and cell death. When administered to young mice the compound reduced glycosphingolipid production 50%-70% in the liver and lymphoid organs without any overt pathology. It is a potent inhibitor of HIV virus replication and syncytium formation in vitro. The compound has been observed to restore cAMP-activated chloride currents in epithelial cystic fibrosis cells through prevented interaction among calnexin and delF508-CFTR. In a mice model for Tay Sachs disease, mice exposed to N-ButyldeoxynojirimycinHCl limited the biosynthesis of GM2 thus preventing accumulation of GM2 in the brain and glycosphingolipids. This action markedly reduced the number of storage neurons and gangliosides per cell. N-ButyldeoxynojirimycinHCl has also rescued trafficking-deficient F508del-CTFR in human airway epithelial cells via inhibition of ER-glucosidases I and II. Also has broad spectrum antiviral activity.
