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Mostrando ítems 1-10 de 43
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
Glycoligand-targeted core-shell nanospheres with tunable drug release profiles from calixarene-cyclodextrin heterodimers
(Royal Society of Chemistry, 2014)
Stable core–shell nanospheres self-assemble in water from heterodimers combining a hydrophobic calix[4]arene moiety and a hydrophilic β-cyclodextrin head; their potential to encapsulate and provide sustained release of the ...
Artículo
Deciphering of polycationic carbohydrate based non-viral gene delivery agents by ESI-LTQ-Orbitrap using CID/HCD pairwise tandem mass spectrometry
(Royal Society of Chemistry, 2016)
For almost three decades, gene therapy has been gaining interest to efficiently treat some severe diseases. In such context, the discovery of an efficient non-viral gene carrier to deliver genetic material into targeted ...
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
Cell uptake mechanisms of glycosylated cationic pDNA-cyclodextrin nanoparticles
(Royal Society of Chemistry, 2015)
The incorporation of carbohydrate functional elements in the architecture of polycationic amphiphilic cyclodextrins (paCDs) provides glycosylated paCDs (pGaCDs) that form transfectious nanocomplexes (glycoCDplexes) with ...
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
Conformationally-locked N -glycosides: Exploiting long-range non-glycone interactions in the design of pharmacological chaperones for Gaucher disease
(Elsevier, 2015)
Pyranoid-type glycomimetics having a cis-1,2-fused glucopyranose–2-alkylsulfanyl-1,3-oxazoline (Glc-PSO) structure exhibit an unprecedented specificity as inhibitors of mammalian β-glucosidase. Notably, their inhibitory ...
Artículo
A novel potential nanophototherapeutic based on the assembly of an amphiphilic cationic ß-cyclodextrin and an anionic porphyrin
(John Wiley & Sons, 2017)
The development of cyclodextrin nanoassemblies as useful carriers for photosensitizer drugs (PS) delivery in biological environment is a topic of increasing interest. In this paper, we present a spectroscopic investigation ...
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
Trehalose- and Glucose-Derived Glycoamphiphiles: Small-Molecule and Nanoparticle Toll-Like Receptor 4 (TLR4) Modulators
(American Chemical Society, 2014)
An increasing number of pathologies have been linked to Toll-like receptor 4 (TLR4) activation and signaling, therefore new hit and lead compounds targeting this receptor activation process are urgently needed. We report ...