Pharmacological Chaperones for β-Galactosidase Related to GM1 -Gangliosidosis and Morquio B: Recent Advances

Arnold E Stütz; Martin Thonhofer; Patrick Weber; Andreas Wolfsgruber; Tanja M Wrodnigg

doi:10.1002/tcr.202100269

Pharmacological Chaperones for β-Galactosidase Related to G_M1 -Gangliosidosis and Morquio B: Recent Advances

Chem Rec. 2021 Nov;21(11):2980-2989. doi: 10.1002/tcr.202100269. Epub 2021 Nov 23.

Authors

Arnold E Stütz¹, Martin Thonhofer¹, Patrick Weber¹, Andreas Wolfsgruber¹, Tanja M Wrodnigg¹

Affiliation

¹ Glycogroup, Institute of Chemistry and Technology of Biobased Systems, Graz University of Technology, Stremayrgasse 9, A-8010, Graz, Austria.

PMID: 34816592
DOI: 10.1002/tcr.202100269

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.

Keywords: Carbohydrates; Inhibitors; Lysosomal storage disease; Lysosomal β-Galactosidase; Pharmacological chaperone.

Publication types

Review

MeSH terms

Gangliosidoses*
Gangliosidosis, GM1* / drug therapy
Humans
Lysosomes
Mucopolysaccharidosis IV* / drug therapy
beta-Galactosidase / antagonists & inhibitors*

Substances

beta-Galactosidase

Abstract

Publication types

MeSH terms

Substances

Grants and funding