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Mostrando ítems 1-10 de 29
Artículo
Dynamic Control of the Self-Assembling Properties of Cyclodextrins by the Interplay of Aromatic and Host-Guest Interactions
(Frontiers Media, 2019)
The presence of a doubly-linked naphthylene clip at the O-2I and O-3II positions in the secondary ring of β-cyclodextrin (βCD) derivatives promoted their self-assembly into head-to-head supramolecular dimers in which the ...
Artículo
Pharmacological chaperone therapy for Gaucher disease: A patent review
(Taylor & Francis, 2011)
Introduction: Mutations in the gene encoding for acid β-glucosidase (β-glucocerebrosidase, GlcCerase) are seen in Gaucher disease (GD), which give rise to significant protein misfolding effects and result in progressive ...
Artículo
Mannosyl-coated nanocomplexes from amphiphilic cyclodextrins and pDNA for site-specific gene delivery
(Elsevier, 2011-10)
Fully homogeneous facial amphiphiles consisting in a cyclodextrin (CD) platform onto which a polycationic cluster and a multi-tail hydrophobic moiety have been installed (polycationic amphiphilic CDs; paCDs) self-organized ...
Artículo
Toward a Suitable Structural Analysis of Gene Delivery Carrier Based on Polycationic Carbohydrates by Electron Transfer Dissociation Tandem Mass Spectrometry
(Elsevier, 2016)
Polycationic carbohydrates represent an attractive class of biomolecules for several applications and particularly as non viral gene delivery vectors. In this case, the establishment of structure-biological activity ...
Artículo
Multivalent glycoligands with lectin/enzyme dual specificity: self-deliverable glycosidase regulators
(Royal Society of Chemistry, 2019)
Multivalent mannosides with inherent macrophage recognition abilities, built on β-cyclodextrin, RAFT cyclopeptide or peptide dendrimer cores, trigger selective inhibition of lysosomal β-glucocerebrosidase or α-mannosidase ...
Artículo
A Bicyclic 1-Deoxygalactonojirimycin Derivative as a Novel Pharmacological Chaperone for GM1 Gangliosidosis
(Cell Press, 2013)
Lysosomal β-galactosidase (β-Gal) deficiency causes a group of disorders that include neuronopathic GM1 gangliosidosis and non-neuronopathic Morquio B disease. We have previously proposed the use of small molecule ligands ...
Artículo
Molecular Determinants for Cyclo-oligosaccharide-based Nanoparticle-mediated Effective siRNA Transfection
(Future Medicine Ltd., 2017)
Aim: To study the structural requirements that a cyclooligosaccharide-based nanoparticle must fulfill to be an efficient siRNA transfection vector. Materials & methods: siRNA protection from degradation by RNAses, transfection ...
Artículo
Self-association of a naphthalene-capped-β-cyclodextrin through cooperative strong hydrophobic interactions
(Elsevier, 2011-09-05)
NMR, circular dichroism and fluorescence techniques were used to study the structure in solution of a new β-cyclodextrin derivate in which naphthalene chromophore group is bridged to O(2) and O(3) secondary positions of ...
Artículo
Bicyclic (galacto)nojirimycin analogues as glycosidase inhibitors: Effect of structural modifications in their pharmacological chaperone potential towards β-glucocerebrosidase
(Royal Society of Chemistry (Great Britain), 2011)
A molecular-diversity-oriented approach for the preparation of bicyclic sp2-iminosugar glycomimetics related to nojirimycin and galactonojirimycin is reported. The synthetic strategy takes advantage of the ability of ...
Artículo
Cyclodextrin-mediated Crystallization of Acid β-glucosidase in Complex with Amphiphilic Bicyclic Nojirimycin Analogues
(Royal Society of Chemistry, 2011)
Cyclodextrin-based host-guest chemistry has been exploited to facilitate co-crystallization of recombinant human acid β-glucosidase (β-glucocerebrosidase, GlcCerase) with amphiphilic bicyclic nojirimycin analogues of the ...