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

Arnold Stütz; Martin Simon Thonhofer; Patrick Weber; Andreas Wolfsgruber; Tanja Maria  Wrodnigg

doi:10.1002/tcr.202100269

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

Arnold Stütz, Martin Simon Thonhofer, Patrick Weber, Andreas Wolfsgruber, Tanja Maria Wrodnigg^*

^*Corresponding author for this work

Institute of Chemistry and Technology of Biobased Systems (6430)

Research output: Contribution to journal › Article › peer-review

Abstract

A short survey on selected β-galactosidase inhibitors as potential pharmacological chaperones for GM1-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

Original language	English
Pages (from-to)	2980-2989
Number of pages	10
Journal	The Chemical Record
Volume	21
Issue number	11
DOIs	https://doi.org/10.1002/tcr.202100269
Publication status	Published - Nov 2021

Keywords

Carbohydrates
Inhibitors
Lysosomal storage disease
Lysosomal β-Galactosidase
Pharmacological chaperone

ASJC Scopus subject areas

Materials Chemistry
Chemical Engineering(all)
Chemistry(all)
Biochemistry

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1002/tcr.202100269Licence: CC BY 4.0

Cite this

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

abstract = "A short survey on selected β-galactosidase inhibitors as potential pharmacological chaperones for GM1-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",

author = "Arnold St{\"u}tz and Thonhofer, {Martin Simon} and Patrick Weber and Andreas Wolfsgruber and Wrodnigg, {Tanja Maria}",

year = "2021",

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doi = "10.1002/tcr.202100269",

language = "English",

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AU - Stütz, Arnold

AU - Thonhofer, Martin Simon

AU - Weber, Patrick

AU - Wolfsgruber, Andreas

AU - Wrodnigg, Tanja Maria

PY - 2021/11

Y1 - 2021/11

N2 - A short survey on selected β-galactosidase inhibitors as potential pharmacological chaperones for GM1-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

AB - A short survey on selected β-galactosidase inhibitors as potential pharmacological chaperones for GM1-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

KW - Carbohydrates

KW - Inhibitors

KW - Lysosomal storage disease

KW - Lysosomal β-Galactosidase

KW - Pharmacological chaperone

UR - http://www.scopus.com/inward/record.url?scp=85119882686&partnerID=8YFLogxK

U2 - 10.1002/tcr.202100269

DO - 10.1002/tcr.202100269

M3 - Article

SN - 1527-8999

VL - 21

SP - 2980

EP - 2989

JO - The Chemical Record

JF - The Chemical Record

IS - 11

ER -

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

Abstract

Keywords

ASJC Scopus subject areas

UN SDGs

Access to Document

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