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.File | Dimensione | Formato | |
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