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Mostrando ítems 1-10 de 17
Artículo
sp2-Iminosugars targeting human lysosomal β-hexosaminidase as pharmacological chaperone candidates for late-onset Tay-Sachs disease
(Taylor & Francis, 2022)
The late-onset form of Tay-Sachs disease displays when the activity levels of human β-hexosaminidase A (HexA) fall below 10% of normal, due to mutations that destabilise the native folded form of the enzyme and impair its ...
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
Mechanocatalytic Depolymerization of Cellulose With Perfluorinated Sulfonic Acid Ionomers
(Frontiers Media, 2018)
Here, we investigated that the mechanocatalytic depolymerization of cellulose in the presence of Aquivion, a sulfonated perfluorinated ionomer. Under optimized conditions, yields of water soluble sugars of 90-97% were ...
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
Harmonized tuning of nucleic acid and lectin binding properties with multivalent cyclodextrins for macrophage-selective gene delivery
(Royal Society of Chemistry, 2015)
Polycationic amphiphilic cyclodextrins (paCDs) have been shown to behave as efficient non-viral gene carriers paralleling the efficacy of commercial vectors towards a variety of cell lines. Their molecular framework and ...
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 ...
Artículo
Glycomimetic-based pharmacological chaperones for lysosomal storage disorders: lessons from Gaucher, GM1-gangliosidosis and Fabry diseases
(Royal Society of Chemistry, 2016)
Lysosomal storage disorders (LSDs) are often caused by mutations that destabilize native folding and impair the trafficking of enzymes, leading to premature endoplasmic reticulum (ER)-associated degradation, deficiencies ...
Artículo
Fluoro-labelled sp2-iminoglycolipids with immunomodulatory properties
(Elsevier, 2023)
The unique electronic properties of the fluorine atom make its strategic incorporation into a bioactive compound a very useful tool in the design of drugs with optimized pharmacological properties. In the field of the ...
Artículo
Synthesis of β-galactosylamides as Ligands of the Peanut Lectin. Insights into the Recognition Process
(Elsevier, 2017)
The synthesis of mono and divalent β-galactosylamides linked to a hydroxylated chain having a C2 symmetry axis derived from L-tartaric anhydride is reported. Reference compounds devoid of hydroxyl groups in the linker were ...