Probing the Inhibitor versus Chaperone Properties of sp2-Iminosugars towards Human β-Glucocerebrosidase: A Picomolar Chaperone for Gaucher Disease

Amphiphilic glycoconjugates as potential anti-cancer chemotherapeutics

(2018) Mugunthan Govindarajan   EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

The sp 2 -iminosugar glycolipid 1-dodecylsulfonyl-5 N ,6 O -oxomethylidenenojirimycin (DSO 2 -ONJ) as selective anti-inflammatory agent by modulation of hemeoxygenase-1 in Bv.2 microglial cells and retinal explants

(2018) Elena Alcalde-Estévez et al.   FOOD AND CHEMICAL TOXICOLOGY

Tuning protein folding in lysosomal storage diseases: the chemistry behind pharmacological chaperones

(2018) David M. Pereira et al.   Chemical Science

A Review of Gaucher Disease Pathophysiology, Clinical Presentation and Treatments

(2017) Jérôme Stirnemann et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Reduction of podocyte globotriaosylceramide content in adult male patients with Fabry disease with amenable GLA mutations following 6 months of migalastat treatment

(2017) Michael Mauer et al.   JOURNAL OF MEDICAL GENETICS

Fluorinated Chaperone−β-Cyclodextrin Formulations for β-Glucocerebrosidase Activity Enhancement in Neuronopathic Gaucher Disease

(2017) M. Isabel García-Moreno et al.   JOURNAL OF MEDICINAL CHEMISTRY

Discovering protein−ligand chalcogen bonding in the protein data bank using endocyclic sulfur-containing heterocycles as ligand search subsets

(2017) Miguel O. Mitchell   JOURNAL OF MOLECULAR MODELING

Drug development in the era of precision medicine

(2017) Sarah A. Dugger et al.   NATURE REVIEWS DRUG DISCOVERY

Orthoester functionalized N-guanidino derivatives of 1,5-dideoxy-1,5-imino-d-xylitol as pH-responsive inhibitors of β-glucocerebrosidase

(2017) Alen Sevšek et al.   MedChemComm

Multivalency as an action principle in multimodal lectin recognition and glycosidase inhibition: a paradigm shift driven by carbon-based glyconanomaterials

(2017) Carmen Ortiz Mellet et al.   Journal of Materials Chemistry B

Protein misfolding in neurodegenerative diseases: implications and strategies

(2017) Patrick Sweeney et al.   Translational Neurodegeneration

Pharmacological Chaperones: Design and Development of New Therapeutic Strategies for the Treatment of Conformational Diseases

(2016) Marino Convertino et al.   ACS Chemical Biology

Modulation of microglia polarization dynamics during diabetic retinopathy in db / db mice

(2016) Ana I. Arroba et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

The Staudinger/aza-Wittig/Grignard reaction as key step for the concise synthesis of 1-C-Alkyl-iminoalditol glycomimetics

(2016) Manuel Zoidl et al.   CARBOHYDRATE RESEARCH

Influence of the configurational pattern of sp2-iminosugar pseudo N-, S-, O- and C-glycosides on their glycoside inhibitory and antitumor properties

(2016) Elena M. Sánchez-Fernández et al.   CARBOHYDRATE RESEARCH

Glycomimetic-based pharmacological chaperones for lysosomal storage disorders: lessons from Gaucher, GM1-gangliosidosis and Fabry diseases

(2016) Elena M. Sánchez-Fernández et al.   CHEMICAL COMMUNICATIONS

Potent Glycosidase Inhibition with Heterovalent Fullerenes: Unveiling the Binding Modes Triggering Multivalent Inhibition

(2016) Marta Abellán Flos et al.   CHEMISTRY-A EUROPEAN JOURNAL

Migalastat: First Global Approval

(2016) Anthony Markham   DRUGS

Efficient stereoselective synthesis of 2-acetamido-1,2-dideoxyallonojirimycin (DAJNAc) and sp 2 -iminosugar conjugates: Novel hexosaminidase inhibitors with discrimination capabilities between the mature and precursor forms of the enzyme

(2016) Alex de la Fuente et al.   EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Inhibitor versus chaperone behaviour of d -fagomine, DAB and LAB sp 2 -iminosugar conjugates against glycosidases: A structure–activity relationship study in Gaucher fibroblasts

(2016) Teresa Mena-Barragán et al.   EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat

(2016) Elfrida R. Benjamin et al.   GENETICS IN MEDICINE

Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study

(2016) Derralynn A Hughes et al.   JOURNAL OF MEDICAL GENETICS

Exploring the Chemistry of Bicyclic Isoxazolidines for the Multicomponent Synthesis of Glycomimetic Building Blocks

(2016) Jorin Hoogenboom et al.   JOURNAL OF ORGANIC CHEMISTRY

Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat

(2016) Dominique P. Germain et al.   NEW ENGLAND JOURNAL OF MEDICINE

Bicyclic isoureas derived from 1-deoxynojirimycin are potent inhibitors of β-glucocerebrosidase

(2016) Alen Sevšek et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Tn Antigen Mimics Based on sp2-Iminosugars with Affinity for an anti-MUC1 Antibody

(2016) Elena M. Sánchez Fernández et al.   ORGANIC LETTERS

pH-Responsive Pharmacological Chaperones for Rescuing Mutant Glycosidases

(2015) Teresa Mena-Barragán et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

A Survey of the Role of Noncovalent Sulfur Interactions in Drug Design

(2015) Brett R. Beno et al.   JOURNAL OF MEDICINAL CHEMISTRY

Fluorescence-Quenched Substrates for Live Cell Imaging of Human Glucocerebrosidase Activity

(2015) Anuj K. Yadav et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders

(2015) Giancarlo Parenti et al.   MOLECULAR THERAPY

Direct synthesis of imino-C-nucleoside analogues and other biologically active iminosugars

(2015) Milan Bergeron-Brlek et al.   Nature Communications

Pharmacological Chaperones and Coenzyme Q10 Treatment Improves Mutant β-Glucocerebrosidase Activity and Mitochondrial Function in Neuronopathic Forms of Gaucher Disease

(2015) Mario de la Mata et al.   Scientific Reports

Molecular Basis of 1-Deoxygalactonojirimycin Arylthiourea Binding to Human α-Galactosidase A: Pharmacological Chaperoning Efficacy on Fabry Disease Mutants

(2014) Yi Yu et al.   ACS Chemical Biology

Structural Basis of Pharmacological Chaperoning for Human β-Galactosidase

(2014) Hironori Suzuki et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Synthesis of Multibranched Australine Derivatives from Reducing Castanospermine Analogues through the Amadori Rearrangement of gem-Diamine Intermediates: Selective Inhibitors of β-Glucosidase

(2014) Elena M. Sánchez-Fernández et al.   JOURNAL OF ORGANIC CHEMISTRY

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

(2014) Julio Rodríguez-Lavado et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Searching for Glycomimetics That Target Protein Misfolding in Rare Diseases: Successes, Failures, and Unexpected Progress Made in Organic Synthesis

(2014) Philippe Compain   SYNLETT

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

(2013) Pilar Alfonso et al.   CHEMBIOCHEM

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

Glucosylceramide Mimics: Highly Potent GCase Inhibitors and Selective Pharmacological Chaperones for Mutations Associated with Types 1 and 2 Gaucher Disease

(2013) Wojciech Schönemann et al.   ChemMedChem

N-Thiocarbonyl Iminosugars: Synthesis and Evaluation of Castanospermine Analogues Bearing Oxazole-2(3H)-thione Moieties

(2013) Sandrina Silva et al.   EUROPEAN JOURNAL OF ORGANIC 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

Stereoselective synthesis of 1-C-alkyl iminogalactitol derivatives, potential chaperones for galactosidase-linked LSDs: a real challenge

(2013) Anna Biela-Banaś et al.   TETRAHEDRON LETTERS

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

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

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

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

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

Functionally Important Aromatic–Aromatic and Sulfur−π Interactions in the D2 Dopamine Receptor

(2012) Kristina N.-M. Daeffler et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Pharmacological chaperone therapy for Gaucher disease: a patent review

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

Iminosugars as therapeutic agents: recent advances and promising trends

(2011) Robert J Nash et al.   Future Medicinal Chemistry

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

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

Fluorescent-tagged sp2-iminosugars with potent β-glucosidase inhibitory activity

(2010) Matilde Aguilar-Moncayo et al.   BIOORGANIC & MEDICINAL CHEMISTRY

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

(2010) Zhuo Luan  et al.   CHEMBIOCHEM

Synthesis of N-, S-, and C-glycoside castanospermine analogues with selective neutral α-glucosidase inhibitory activity as antitumour agents

(2010) Elena M. Sánchez-Fernández et al.   CHEMICAL COMMUNICATIONS

Iminosugars past, present and future: medicines for tomorrow

(2010) Graeme Horne et al.   DRUG DISCOVERY TODAY

Synthesis and Evaluation of Lipophilic Aza-C-glycosides as Inhibitors of Glucosylceramide Metabolism

(2010) Tom Wennekes et al.   EUROPEAN JOURNAL OF ORGANIC CHEMISTRY

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

(2009) Zhuo Luan et al.   CHEMBIOCHEM

6-Amino-6-deoxy-5,6-di-N-(N′-octyliminomethylidene)nojirimycin: Synthesis, Biological Evaluation, and Crystal Structure in Complex with Acid β-Glucosidase

(2009) Boris Brumshtein et al.   CHEMBIOCHEM

Synthesis of Thiohydantoin-Castanospermine Glycomimetics as Glycosidase Inhibitors

(2009) Matilde Aguilar-Moncayo et al.   JOURNAL OF ORGANIC CHEMISTRY

The Pharmacological Chaperone 1-Deoxygalactonojirimycin Reduces Tissue Globotriaosylceramide Levels in a Mouse Model of Fabry Disease

(2009) Richie Khanna et al.   MOLECULAR THERAPY

From carbohydrate leads to glycomimetic drugs

(2009) Beat Ernst et al.   NATURE REVIEWS DRUG DISCOVERY

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

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

Generalized Anomeric Effect ingem-Diamines: Stereoselective Synthesis of α-N-Linked Disaccharide Mimics

(2009) Elena M. Sánchez-Fernández et al.   ORGANIC LETTERS

Synthesis and evaluation of sulfamide-type indolizidines as glycosidase inhibitors

(2008) Mahmoud Benltifa et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Molecular Basis for β-Glucosidase Inhibition by Ring-Modified Calystegine Analogues

(2008) Matilde Aguilar et al.   CHEMBIOCHEM

Synthesis and Biological Evaluation of Guanidine-Type Iminosugars

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

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

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