Issue 69, 2016, Issue in Progress
From the journal:

RSC Advances

Evidence for a multivalent effect in inhibition of sulfatases involved in lysosomal storage disorders (LSDs)†

G. D'Adamio,a   C. Matassini,*a   C. Parmeggiani,ab   S. Catarzi,c   A. Morrone,c   A. Goti,a   P. Paolid  and  F. Cardona*a  

* Corresponding authors

a Department of Chemistry “Ugo Schiff”, University of Florence, Via della Lastruccia 3-13, 50019 Sesto Fiorentino, FI, Italy
E-mail: francesca.cardona@unifi.it, camilla.matassini@unifi.it

b CNR – INO and LENS, via N. Carrara 1, 50019 Sesto Fiorentino, FI, Italy

c Paediatric Neurology Unit and Laboratories, Neuroscience Department, Meyer Children's Hospital, Department of Neurosciences, Pharmacology and Child Health, University of Florence, Viale Pieraccini n. 24, 50139 Firenze, Italy

d Department of Experimental and Clinical Biomedical Sciences, University of Florence, Viale Morgagni 50, 50134 Florence, Italy

Abstract

Two newly synthesized nonavalent polyhydroxylated pyrrolidine iminosugars are the first examples of multivalent inhibitors of GALNS and IDS lysosomal enzymes, whose deficiency leads to Morquio A syndrome and Hunter disease, respectively, and pave the way to a pharmacological chaperone or stabilizing enzyme therapy for these LSDs.

Graphical abstract: Evidence for a multivalent effect in inhibition of sulfatases involved in lysosomal storage disorders (LSDs)

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Article information

DOI
https://doi.org/10.1039/C6RA15806D
Article type
Communication
Submitted
17 Jun 2016
Accepted
29 Jun 2016
First published
01 Jul 2016

RSC Adv., 2016,6, 64847-64851

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Evidence for a multivalent effect in inhibition of sulfatases involved in lysosomal storage disorders (LSDs)

G. D'Adamio, C. Matassini, C. Parmeggiani, S. Catarzi, A. Morrone, A. Goti, P. Paoli and F. Cardona, RSC Adv., 2016, 6, 64847 DOI: 10.1039/C6RA15806D

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