1,6-<i>epi</i>-Cyclophellitol Cyclosulfamidate Is a Bona Fide Lysosomal α-Glucosidase Stabilizer for the Treatment of Pompe Disease-Reference-Cited by-同舟云学术

1,6-epi-Cyclophellitol Cyclosulfamidate Is a Bona Fide Lysosomal α-Glucosidase Stabilizer for the Treatment of Pompe Disease

Published:2022-08-02 Issue:32 Volume:144 Page:14819-14827
ISSN:0002-7863
Container-title:Journal of the American Chemical Society
language:en
Short-container-title:J. Am. Chem. Soc.

Author:

Kok Ken¹,Kuo Chi-Lin¹^ORCID,Katzy Rebecca E.¹,Lelieveld Lindsey T.¹,Wu Liang²^ORCID,Roig-Zamboni Véronique³,van der Marel Gijsbert A.⁴,Codée Jeroen D. C.⁴^ORCID,Sulzenbacher Gerlind³^ORCID,Davies Gideon J.²^ORCID,Overkleeft Herman S.⁴^ORCID,Aerts Johannes M. F. G.¹^ORCID,Artola Marta¹^ORCID

Affiliation:

1. Department of Medical Biochemistry, Leiden Institute of Chemistry, Leiden University, Einsteinweg 55, Leiden 2333 CC, The Netherlands

2. Department of Chemistry, University of York, York YO10 5DD, U.K.

3. Architecture et Fonction des Macromolécules Biologiques (AFMB), CNRS, Aix-Marseille University, Marseille 13288, France

4. Department of Bio-Organic Synthesis, Leiden Institute of Chemistry, Leiden University, Einsteinweg 55, Leiden 2333 CC, The Netherlands

Funder

Royal Society

Sanofi

French Infrastructure for Integrated Structural Biology

Centre National de la Recherche Scientifique

H2020 European Research Council

Nederlandse Organisatie voor Wetenschappelijk Onderzoek