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Mostrando ítems 1-10 de 36
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
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 ...
Tesis Doctoral
Plataformas multifuncionales basadas en ciclodextrinas: diseño de antitoxinas del ántrax y vectores de genes
(2010-02-12)
El trabajo desarrollado en esta Tesis aprovecha precisamente la faceta como plataforma nanométrica de las CDs. En ella se ha abordado la elaboración de estructuras molecularmente bien definidas basadas en CDs capaces de ...
Tesis Doctoral
Síntesis de iminoazúcares sp2 y evaluación de su actividad como chaperonas farmacológicas para el tratamiento de las enfermedades de Gaucher, Fabry y gangliosidosis GM1
(2013-12-13)
Las enfermedades de depósito lisosomal (Lysosomal Storage Disorders, LSDs), entre las que se encuentran la enfermedad de Gaucher (GD), la enfermedad de Fabry (FD) y la gangliosidosis GM1 (GM1) son un conjunto de enfermedades ...
Artículo
Probing the Inhibitor versus Chaperone Properties of sp2-Iminosugars towards Human -Glucocerebrosidase: A Picomolar Chaperone for Gaucher Disease
(Molecular Diversity Preservation International, 2018)
A series of sp2-iminosugar glycomimetics differing in the reducing or nonreducing character, the configurational pattern (d-gluco or l-ido), the architecture of the glycone skeleton, and the nature of the nonglycone ...
Tesis Doctoral
Artículo
Novel Therapies for Orphan Diseases
(American Chemical Society, 2019)
"Orphan" does not mean infrequent: over 7000 rare diseases affect millions of individuals. The US Orphan Drug Act and analogous regulations have succeeded at accelerating the development of novel therapies, but high prices ...
Patente. Invención
Ciclooligosacáridos anfifílicos policatiónicos y su uso como transportadores moleculares
(Oficina Española de Patentes y Marcas (OEPM), 2011-09-14)
Ciclooligosacáridos anfifílicos policatiónicos y su uso como transportadores moleculares. La presente invención se refiere a un grupo de ciclooligosacáridos anfifílicos policatiónicos que, debido a su estructura molecular, ...
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 ...