A Selective Inhibitor Gal-PUGNAc of Human Lysosomal beta-Hexosaminidases Modulates Levels of the Ganglioside GM2 in Neuroblastoma Cells

摘要

Imbalances in the levels of many glycoconjugates have been implicated in human diseases.Many inhibitors of the large super family of glycoside hydrolases,which are the enzymes that break down these glycoconjugates,are known.However,few of these inhibitors have been developed and characterized as being able to selectively target the enzyme of interest over other functionally related glycoside hydrolases.Such highly selective inhibitors are critical tools to gain insights into the roles of specific glycoconjugates in disease etiology and normal cellular function.