Article
pH-Responsive Pharmacological Chaperones for Rescuing Mutant Glycosidases.
Angewandte Chemie (International ed. in English) - 28 Sept 2015
Mena-Barragán Teresa, Narita Aya, Matias Dino, Tiscornia Gustavo, Nanba Eiji, Ohno Kousaku, Suzuki Yoshiyuki, Higaki Katsumi, Garcia Fernández José Manuel, Ortiz Mellet Carmen
Abstract excerpt
A general approach is reported for the design of small-molecule competitive inhibitors of lysosomal glycosidases programmed to 1) promote correct folding of mutant enzymes at the endoplasmic reticulum, 2) facilitate trafficking, and 3) undergo dissociation and self-inactivation at the lysosome. The strategy is based on the incorporation of an orthoester segment into iminosugar conjugates to switch the nature of...
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