Design of multivalent pharmacological chaperones against Pompe disease via metal-free ligation

Organic & Biomolecular Chemistry 2026 Open access

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Authors: Marc Borie-Guichot, Abdelouahd Oukhrib, My Lan Tran, Cédric‐Olivier Turrin, Yves Génisson, Laure Gibot, Stéphanie Ballereau, Cécile Dehoux-Baudoin
DOI: 10.1039/d6ob00021e
Cited by: 1
References: 42

Abstract

-undecanyl-DNJ derivative, was found to enhance GAA activity by 1.2 fold in fibroblasts from a Pompe patient at the dose of only 0.5 nM. This finding is a significant breakthrough as it represents the first report of a multivalent compound increasing GAA activity in patient cells. This novel multivalent PC, which is a very weak GAA inhibitor, offers a promising avenue for the development of effective treatments for Pompe disease.

Fields of Study & Subjects

Lysosomal Storage Disorders Research Calcium signaling and nucleotide metabolism Glycogen Storage Diseases and Myoclonus

Publication details

Journal / source
Organic & Biomolecular Chemistry
Publisher
Royal Society of Chemistry (RSC)
Volume
24
Issue
12
Pages
2487–2497
Publication date
1 January 2026
Language
EN