FURANOID CARBASUGARS AS GH 20 HEXOSAMINIDASE INHIBITORS AND POTENTIAL PHARMACOLOGICAL CHAPERONS

摘要

Lysosomal storage diseases (LSDs) are a series of metabolic disorders of lysosomal enzymes caused by mutations of specific genes, responsible for their biosynthesis. Roughly, 25 % of these enzymes are involved in the chemical manipulation of N-acetyl-D-glucosaminyl or N-acetyl-D-galactosaminyl residues from degradation-bound polysaccharides and glycoconjugates [1].