Targeted delivery of pharmacological chaperones for Gaucher disease to macrophages by a mannosylated cyclodextrin carrier

In vivo performance of polymer nanocarriers dually-targeted to epitopes of the same or different receptors

(2013) Iason T. Papademetriou et al.   BIOMATERIALS

Chaperone therapy update: Fabry disease, GM1-gangliosidosis and Gaucher disease

(2013) Yoshiyuki Suzuki   BRAIN & DEVELOPMENT

Bicyclic Derivatives ofL-Idonojirimycin as Pharmacological Chaperones for Neuronopathic Forms of Gaucher Disease

(2013) Pilar Alfonso et al.   CHEMBIOCHEM

Recent developments in design and synthesis of bicyclic azasugars, carbasugars and related molecules as glycosidase inhibitors

(2013) Rima Lahiri et al.   CHEMICAL SOCIETY REVIEWS

Cyclodextrin-based multivalent glycodisplays: covalent and supramolecular conjugates to assess carbohydrate–protein interactions

(2013) Álvaro Martínez et al.   CHEMICAL SOCIETY REVIEWS

Multivalent glycocalixarenes for recognition of biological macromolecules: glycocalyx mimics capable of multitasking

(2013) Francesco Sansone et al.   CHEMICAL SOCIETY REVIEWS

Multivalent glycoconjugate syntheses and applications using aromatic scaffolds

(2013) Yoann M. Chabre et al.   CHEMICAL SOCIETY REVIEWS

Fullerene-sp2-Iminosugar Balls as Multimodal Ligands for Lectins and Glycosidases: A Mechanistic Hypothesis for the Inhibitory Multivalent Effect

(2013) Rocío Rísquez-Cuadro et al.   CHEMISTRY-A EUROPEAN JOURNAL

Glucocerebrosidase inhibitors for the treatment of Gaucher disease

(2013) Ana Trapero et al.   Future Medicinal Chemistry

Pharmacological Chaperones as Therapeutics for Lysosomal Storage Diseases

(2013) Robert E. Boyd et al.   JOURNAL OF MEDICINAL CHEMISTRY

A Bicyclic 1-Deoxygalactonojirimycin Derivative as a Novel Pharmacological Chaperone for GM1 Gangliosidosis

(2013) Tomoko Takai et al.   MOLECULAR THERAPY

New Castanospermine Glycoside Analogues Inhibit Breast Cancer Cell Proliferation and Induce Apoptosis without Affecting Normal Cells

(2013) Ghada Allan et al.   PLoS One

Adamantane substituted aminocyclitols as pharmacological chaperones for Gaucher disease

(2013) Ana Trapero et al.   MedChemComm

Enzyme Replacement Therapy for Lysosomal Diseases: Lessons from 20 Years of Experience and Remaining Challenges

(2012) R.J. Desnick et al.   Annual Review of Genomics and Human Genetics

Functional Screening of Pharmacological Chaperones via Restoration of Enzyme Activity upon Denaturation

(2012) Meera Shanmuganathan et al.   BIOCHEMISTRY

Cyclodextrin-Based Iminosugar Click Clusters: The First Examples of Multivalent Pharmacological Chaperones for the Treatment of Lysosomal Storage Disorders

(2012) Camille Decroocq et al.   CHEMBIOCHEM

Tuning glycosidase inhibition through aglycone interactions: pharmacological chaperones for Fabry disease and GM1 gangliosidosis

(2012) M. Aguilar-Moncayo et al.   CHEMICAL COMMUNICATIONS

Multivalent glycoconjugates as anti-pathogenic agents

(2012) Anna Bernardi et al.   CHEMICAL SOCIETY REVIEWS

Multivalency in heterogeneous glycoenvironments: hetero-glycoclusters, -glycopolymers and -glycoassemblies

(2012) José L. Jiménez Blanco et al.   CHEMICAL SOCIETY REVIEWS

Multivalent glyco(cyclo)peptides

(2012) M. Carmen Galan et al.   CHEMICAL SOCIETY REVIEWS

Sweet carbon nanostructures: carbohydrate conjugates with carbon nanotubes and graphene and their applications

(2012) Yanan Chen et al.   CHEMICAL SOCIETY REVIEWS

sp2-Iminosugar O-, S-, and N-Glycosides as Conformational Mimics of α-Linked Disaccharides; Implications for Glycosidase Inhibition

(2012) Elena M. Sánchez-Fernández et al.   CHEMISTRY-A EUROPEAN JOURNAL

Neuronopathic Gaucher's disease: induced pluripotent stem cells for disease modelling and testing chaperone activity of small compounds

(2012) Gustavo Tiscornia et al.   HUMAN MOLECULAR GENETICS

Lysosomal storage disorders: The cellular impact of lysosomal dysfunction

(2012) Frances M. Platt et al.   JOURNAL OF CELL BIOLOGY

The mannose receptor

(2012) Luisa Martinez-Pomares   JOURNAL OF LEUKOCYTE BIOLOGY

Discovery, Structure–Activity Relationship, and Biological Evaluation of Noninhibitory Small Molecule Chaperones of Glucocerebrosidase

(2012) Samarjit Patnaik et al.   JOURNAL OF MEDICINAL CHEMISTRY

Conformationally-Locked N-Glycosides with Selective β-Glucosidase Inhibitory Activity: Identification of a New Non-Iminosugar-Type Pharmacological Chaperone for Gaucher Disease

(2012) Javier Castilla et al.   JOURNAL OF MEDICINAL CHEMISTRY

Potent Aminocyclitol Glucocerebrosidase Inhibitors are Subnanomolar Pharmacological Chaperones for Treating Gaucher Disease

(2012) Ana Trapero et al.   JOURNAL OF MEDICINAL CHEMISTRY

Rapid Assembly of a Library of Lipophilic Iminosugars via the Thiol–Ene Reaction Yields Promising Pharmacological Chaperones for the Treatment of Gaucher Disease

(2012) Ethan D. Goddard-Borger et al.   JOURNAL OF MEDICINAL CHEMISTRY

Binding of 3,4,5,6-Tetrahydroxyazepanes to the Acid-β-glucosidase Active Site: Implications for Pharmacological Chaperone Design for Gaucher Disease

(2011) Susan D. Orwig et al.   BIOCHEMISTRY

Mannosyl-coated nanocomplexes from amphiphilic cyclodextrins and pDNA for site-specific gene delivery

(2011) Alejandro Díaz-Moscoso et al.   BIOMATERIALS

Second-Generation Iminoxylitol-Based Pharmacological Chaperones for the Treatment of Gaucher Disease

(2011) Farah Oulaïdi et al.   ChemMedChem

Pharmacological chaperone therapy for Gaucher disease: a patent review

(2011) Juan M Benito et al.   EXPERT OPINION ON THERAPEUTIC PATENTS

Probing Carbohydrate-Lectin Recognition in Heterogeneous Environments with Monodisperse Cyclodextrin-Based Glycoclusters

(2011) Marta Gómez-García et al.   JOURNAL OF ORGANIC CHEMISTRY

Copper(II)-Complex Directed Regioselective Mono-p-Toluenesulfonylation of Cyclomaltoheptaose at a Primary Hydroxyl Group Position: An NMR and Molecular Dynamics-Aided Design

(2011) Ho Law et al.   JOURNAL OF PHYSICAL CHEMISTRY B

Polyhydroxylated Bicyclic Isoureas and Guanidines Are Potent Glucocerebrosidase Inhibitors and Nanomolar Enzyme Activity Enhancers in Gaucher Cells

(2011) Ana Trapero et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Enhanced delivery of α-glucosidase for Pompe disease by ICAM-1-targeted nanocarriers: comparative performance of a strategy for three distinct lysosomal storage disorders

(2011) Janet Hsu et al.   Nanomedicine-Nanotechnology Biology and Medicine

Bicyclic (galacto)nojirimycin analogues as glycosidase inhibitors: Effect of structural modifications in their pharmacological chaperone potential towards β-glucocerebrosidase

(2011) Matilde Aguilar-Moncayo et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Cyclodextrin-mediated crystallization of acid β-glucosidase in complex with amphiphilic bicyclic nojirimycin analogues

(2011) Boris Brumshtein et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Synthesis and glycosidase inhibitory activity of isourea-type bicyclic sp2-iminosugars related to galactonojirimycin and allonojirimycin

(2011) Matilde Aguilar-Moncayo et al.   TETRAHEDRON

(Pseudo)amide-linked oligosaccharide mimetics: molecular recognition and supramolecular properties

(2010) José L Jiménez Blanco et al.   Beilstein Journal of Organic Chemistry

A Fluorescent sp2-Iminosugar With Pharmacological Chaperone Activity for Gaucher Disease: Synthesis and Intracellular Distribution Studies

(2010) Zhuo Luan  et al.   CHEMBIOCHEM

Thermodynamics and chemical characterization of protein–carbohydrate interactions: The multivalency issue

(2010) Michael Reynolds et al.   COMPTES RENDUS CHIMIE

Pharmacological small molecules for the treatment of lysosomal storage disorders

(2010) BE Smid et al.   EXPERT OPINION ON INVESTIGATIONAL DRUGS

Enhanced endothelial delivery and biochemical effects of α-galactosidase by ICAM-1-targeted nanocarriers for Fabry disease

(2010) Janet Hsu et al.   JOURNAL OF CONTROLLED RELEASE

Comparative studies on lectin–carbohydrate interactions in low and high density homo- and heteroglycoclusters

(2010) Marta Gómez-García et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Design, synthesis and biological evaluation of carbohydrate-functionalized cyclodextrins and liposomes for hepatocyte-specific targeting

(2010) Gonçalo J. L. Bernardes et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Glycosphingolipids-Nature, Function, and Pharmacological Modulation

(2009) Tom Wennekes et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Chaperone Activity of Bicyclic Nojirimycin Analogues for Gaucher Mutations in Comparison withN-(n-nonyl)Deoxynojirimycin

(2009) Zhuo Luan et al.   CHEMBIOCHEM

Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics

(2009) Giancarlo Parenti   EMBO Molecular Medicine

Glycosidase inhibition by ring-modified castanospermine analogues: tackling enzyme selectivity by inhibitor tailoring

(2009) Matilde Aguilar-Moncayo et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Substrate reduction therapy

(2008) Frances M Platt et al.   ACTA PAEDIATRICA

Dendritic Cell Interaction withCandida albicansCritically Depends onN-Linked Mannan

(2008) Alessandra Cambi et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Synthesis and Biological Evaluation of Guanidine-Type Iminosugars

(2008) Matilde Aguilar et al.   JOURNAL OF ORGANIC CHEMISTRY

Phenotype, diagnosis, and treatment of Gaucher's disease

(2008) Gregory A Grabowski   LANCET

The Carbohydrate-Active EnZymes database (CAZy): an expert resource for Glycogenomics

(2008) B. L. Cantarel et al.   NUCLEIC ACIDS RESEARCH

A counterintuitive approach to treat enzyme deficiencies: use of enzyme inhibitors for restoring mutant enzyme activity

(2007) Jian-Qiang Fan   BIOLOGICAL CHEMISTRY