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

Michael Schalli, Patrick Weber, Christina Tysoe, Bettina M. Pabst, Martin Thonhofer, Eduard Paschke, Arnold Stütz, Marion Tschernutter, Werner Windischhofer, Stephen G Withers

Research output: Contribution to journalArticlepeer-review

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.
Original languageEnglish
Pages (from-to)3431-3435
JournalBioorganic & Medicinal Chemistry Letters
Volume27
Issue number15
Publication statusPublished - 2017

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