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.

Evidence for a multivalent effect in inhibition of sulfatases involved in lysosomal storage disorders (LSDs) / D'Adamio, Giampero; Matassini, Camilla; Parmeggiani, Camilla; Catarzi, Serena; Morrone, Amelia; Goti, Andrea; Paoli, Paolo; Cardona, Francesca. - In: RSC ADVANCES. - ISSN 2046-2069. - STAMPA. - 6:(2016), pp. 64847-64851. [10.1039/c6ra15806d]

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

MATASSINI, CAMILLA;PARMEGGIANI, CAMILLA;CATARZI, SERENA;MORRONE, AMELIA;GOTI, ANDREA;PAOLI, PAOLO;CARDONA, FRANCESCA
2016

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.
2016
6
64847
64851
D'Adamio, Giampero; Matassini, Camilla; Parmeggiani, Camilla; Catarzi, Serena; Morrone, Amelia; Goti, Andrea; Paoli, Paolo; Cardona, Francesca...espandi
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Utilizza questo identificatore per citare o creare un link a questa risorsa: https://hdl.handle.net/2158/1044584
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