Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2017 Aug 1;27(15):3431-3435.
doi: 10.1016/j.bmcl.2017.05.086. Epub 2017 May 30.

A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols

Michael Schalli  1 , Patrick Weber  1 , Christina Tysoe  2 , Bettina M Pabst  3 , Martin Thonhofer  1 , Eduard Paschke  3 , Arnold E Stütz  4 , Marion Tschernutter  3 , Werner Windischhofer  3 , Stephen G Withers  2
Affiliations

A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols

Michael Schalli et al. Bioorg Med Chem Lett. .

Abstract

N-Functionalized amino(hydroxymethyl)cyclopentanetriols are potent inhibitors of β-d-galactosidases and, for the first time, could be shown to act as pharmacological chaperones for GM1-gangliosidosis-associated lysosomal acid β-galactosidase thus representing a new structural type of pharmacological chaperones for this lysosomal storage disease.

Keywords: Aminocyclopentane; Carbafuranose; G(M1)-Gangliosidosis; Galactosidase inhibitor; Pharmacological chaperone.

PubMed Disclaimer

Publication types

LinkOut - more resources