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^*

^*Korrespondierende/r Autor/-in für diese Arbeit

Institut für Chemie und Technologie Biobasierter Systeme (6430)

Publikation: Beitrag in einer Fachzeitschrift › Artikel › Begutachtung

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

Originalsprache	englisch
Seiten (von - bis)	2980-2989
Seitenumfang	10
Fachzeitschrift	The Chemical Record
Jahrgang	21
Ausgabenummer	11
DOIs	https://doi.org/10.1002/tcr.202100269
Publikationsstatus	Veröffentlicht - Nov. 2021

ASJC Scopus subject areas

Werkstoffchemie
Allgemeine chemische Verfahrenstechnik
Allgemeine Chemie
Biochemie

UN SDGs

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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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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

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JO - The Chemical Record

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

Abstract

ASJC Scopus subject areas

UN SDGs

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