α-d-Gal-cyclophellitol cyclosulfamidate is a Michaelis complex analog that stabilizes therapeutic lysosomal α-galactosidase A in Fabry disease-Reference-Cited by-同舟云学术

α-d-Gal-cyclophellitol cyclosulfamidate is a Michaelis complex analog that stabilizes therapeutic lysosomal α-galactosidase A in Fabry disease

Published:2019 Issue:40 Volume:10 Page:9233-9243
ISSN:2041-6520
Container-title:Chemical Science
language:en
Short-container-title:Chem. Sci.

Author:

Artola Marta¹²³⁴⁵^ORCID,Hedberg Christinne¹²³⁴⁵^ORCID,Rowland Rhianna J.⁶⁷⁸⁹^ORCID,Raich Lluís¹⁰¹¹¹²¹³¹⁴,Kytidou Kassiani¹⁵²⁴⁵,Wu Liang⁶⁷⁸⁹,Schaaf Amanda¹²³⁴⁵,Ferraz Maria Joao¹⁵²⁴⁵,van der Marel Gijsbert A.¹²³⁴⁵,Codée Jeroen D. C.¹²³⁴⁵^ORCID,Rovira Carme¹⁰¹¹¹²¹³¹⁴^ORCID,Aerts Johannes M. F. G.¹⁵²⁴⁵^ORCID,Davies Gideon J.⁶⁷⁸⁹^ORCID,Overkleeft Herman S.¹²³⁴⁵^ORCID

Affiliation:

1. Department of Bio-organic Synthesis

2. Leiden Institute of Chemistry

3. Leiden University

4. 2333 CC Leiden

5. The Netherlands

6. Department of Chemistry

7. York Structural Biology Laboratory

8. University of York

9. UK

10. Departament de Química Inorgànica i Orgànica (Secció de Química Orgànica)

11. Institut de Química Teòrica i Computacional (IQTCUB)

12. Universitat de Barcelona

13. 08028 Barcelona

14. Spain

15. Department of Medical Biochemistry

Abstract

α-d-Gal-cyclophellitol cyclosulfamidate is a new class of neutral, conformationally-constrained competitive glycosidase inhibitor that stabilizes α-gal A and prevents its degradation both in vitro and in cellulo by mimicry of the Michaelis complex conformation.

Funder

H2020 European Research Council

Royal Society

Diamond Light Source

Sanofi

Ministerio de Ciencia e Innovación

Biotechnology and Biological Sciences Research Council

Nederlandse Organisatie voor Wetenschappelijk Onderzoek

Generalitat de Catalunya

Fondo Europeo de Desarrollo Regional

Villum Villum Foundation

Publisher