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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
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
Cyclodextrin- and calixarene-based polycationic amphiphiles as gene delivery systems: A structure-activity relationship study
(Royal Society of Chemistry, 2015)
Multi-head/multi-tail facial amphiphiles built on cyclodextrin (CD) and calixarene (CA) scaffolds are paradigmatic examples of monodisperse gene delivery systems. The possibility to precisely control the architectural ...
Artículo
Multiply–linked cyclodextrin–aromatic hybrids: Caps, hinges and clips
(Taylor & Francis, 2019)
The judicious combination of shaping and recognition elements in cage-type architectures represents a powerful strategy to access molecular devices with tailored receptor properties and controlled abilities to form ...
Artículo
Efficient stereoselective synthesis of 2-acetamido-1,2-dideoxyallonojirimycin (DAJNAc) and sp2-iminosugar conjugates: Novel hexosaminidase inhibitors with discrimination capabilities between the mature and precursor forms of the enzyme
(Elsevier, 2016)
Due to their capacity to inhibit hexosaminidases, 2-acetamido-1,2-dideoxy-iminosugars have been widely studied as potential therapeutic agents for various diseases. An efficient stereoselective synthesis of 2-acetamido-1 ...
Artículo
Pharmacological Chaperones for the Treatment of α-Mannosidosis
(American Chemical Society, 2019)
α-Mannosidosis (AM) results from deficient lysosomal α-mannosidase (LAMAN) activity and subsequent substrate accumulation in the lysosome, leading to severe pathology. Many of the AM-causative mutations compromise enzyme ...
Artículo
Carbon Dioxide as a Traceless Caramelization Promotor: Preparation of Prebiotic Difructose Dianhydrides (DFAs)-Enriched Caramels from D-Fructose
(American Chemical Society, 2017)
Activation of a concentrated solution of D-fructose with carbonic acid, generated from carbon dioxide, induces the formation of difructose dianhydrides (DFAs) and their glycosylated derivatives (glycosyl-DFAs), a family ...