Pharmacological Chaperones for β‐Galactosidase Related to G<sub>M1</sub>‐Gangliosidosis and Morquio B: Recent Advances
Arnold Stütz, Martin Thonhofer, Patrick Weber, Andreas Wolfsgruber, Tanja M. Wrodnigg
Abstract
Abstract A short survey on selected β‐galactosidase inhibitors as potential pharmacological chaperones for G M1 ‐gangliosidosis and Morquio B associated mutants of human lysosomal β‐galactosidase is provided highlighting recent developments in this particular area of lysosomal storage disorders and orphan diseases.
Topics & Concepts
GangliosidosisLysosomal storage disordersGalactosidasesBeta-galactosidaseBiologyBiochemistryEnzymeGeneGene expressionLysosomal Storage Disorders ResearchTrypanosoma species research and implicationsCellular transport and secretion