N-(n-Butyl)deoxygalactonojirimycin (CAS 141206-42-0)

N-(n-Butyl)deoxygalactonojirimycin, known as NB-DGJ, is a highly potent and selective inhibitor of α-gal A (a-D-galactosidase). It also acts as a selective inhibitor of GlcCer synthesis. In addition to inhibiting glucosylceramide synthase, N-(n-Butyl)deoxygalactonojirimycin effectively targets glucocerebrosidase (GBA), β-glucosidase 2 (GBA2), acid α-glucosidase, sucrase, maltase, and lactase. By reducing glycolipid levels in HL-60 and WEHI-3B cells at concentrations ranging from 0.5 to 500 µM, N-(n-Butyl)deoxygalactonojirimycin demonstrates its efficacy as a glycolipid biosynthesis inhibitor. Notably, when administered intraperitoneally at doses of 600 and 1,200 mg/kg four times per day for three days, N-(n-Butyl)deoxygalactonojirimycin exhibits a reduction in brain ganglioside levels in a Glb1-/- neonatal mouse model of GM1 gangliosidosis. Additionally, in mice, treatment with N-(n-Butyl)deoxygalactonojirimycin at doses ranging from 1 to 1,200 mg/kg per day for 35 days increases testicular glucosylceramide levels. Furthermore, N-(n-Butyl)deoxygalactonojirimycin, at doses of 15, 25, and 50 mg/kg per day, induces abnormal spermatid and acrosome formation while reducing motility in isolated mouse epididymal sperm. The observed effect of infertility induced by N-(n-Butyl)deoxygalactonojirimycin in the same model can be reversed upon withdrawal.