(Q38797648)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27043200%20AND%20SRC:MED&resulttype=core&format=json

31 March 2020

title

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

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27043200%20AND%20SRC:MED&resulttype=core&format=json

31 March 2020

0 references

Elena M Sánchez Fernández

series ordinal

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27043200%20AND%20SRC:MED&resulttype=core&format=json

José Manuel García Fernández

31 March 2020

series ordinal

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27043200%20AND%20SRC:MED&resulttype=core&format=json

31 March 2020

author name string

Carmen Ortiz Mellet

series ordinal

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27043200%20AND%20SRC:MED&resulttype=core&format=json

31 March 2020

language of work or name

English

0 references

publication date

1 April 2016

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27043200%20AND%20SRC:MED&resulttype=core&format=json

31 March 2020

published in

Chemical Communications

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27043200%20AND%20SRC:MED&resulttype=core&format=json

31 March 2020

volume

52

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27043200%20AND%20SRC:MED&resulttype=core&format=json

31 March 2020

issue

32

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27043200%20AND%20SRC:MED&resulttype=core&format=json

31 March 2020

page(s)

5497-5515

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27043200%20AND%20SRC:MED&resulttype=core&format=json

The cell biology of disease: lysosomal storage disorders: the cellular impact of lysosomal dysfunction

31 March 2020

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A Guided Tour of the Structural Biology of Gaucher Disease: Acid-β-Glucosidase and Saposin C.

21 January 2018

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Degradation of misfolded proteins in neurodegenerative diseases: therapeutic targets and strategies

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Multicenter analysis of glucocerebrosidase mutations in Parkinson's disease.

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

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Ex vivo and in vivo effects of isofagomine on acid β-glucosidase variants and substrate levels in Gaucher disease

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Pharmacological chaperone therapy for Gaucher disease: a patent review.

21 January 2018

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Isofagomine in vivo effects in a neuronopathic Gaucher disease mouse.

21 January 2018

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The iminosugar isofagomine increases the activity of N370S mutant acid beta-glucosidase in Gaucher fibroblasts by several mechanisms

21 January 2018

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Docking and SAR studies of calystegines: binding orientation and influence on pharmacological chaperone effects for Gaucher's disease.

21 January 2018

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Synthesis and glycosidase inhibitory activity of noeurostegine-a new and potent inhibitor of beta-glucoside hydrolases

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

21 January 2018

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Crystal structures of complexes of N-butyl- and N-nonyl-deoxynojirimycin bound to acid beta-glucosidase: insights into the mechanism of chemical chaperone action in Gaucher disease

21 January 2018

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21 January 2018

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21 January 2018

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Rapid modifications of N-substitution in iminosugars: development of new β-glucocerebrosidase inhibitors and pharmacological chaperones for Gaucher disease

21 January 2018

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21 January 2018

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21 January 2018

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Rapid assembly of a library of lipophilic iminosugars via the thiol-ene reaction yields promising pharmacological chaperones for the treatment of Gaucher disease

21 January 2018

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21 January 2018

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

21 January 2018

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21 January 2018

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21 January 2018

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Fullerene-sp2-iminosugar balls as multimodal ligands for lectins and glycosidases: a mechanistic hypothesis for the inhibitory multivalent effect.

21 January 2018

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Influence of the configurational pattern of sp(2)-iminosugar pseudo N-, S-, O- and C-glycosides on their glycoside inhibitory and antitumor properties.

21 January 2018

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Synthesis and evaluation of calystegine B2 analogues as glycosidase inhibitors

21 January 2018

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Castanospermine-trehazolin hybrids: a new family of glycomimetics with tuneable glycosidase inhibitory properties.

21 January 2018

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

21 January 2018

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

21 January 2018

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Synthesis and biological evaluation of guanidine-type iminosugars.

21 January 2018

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Synthesis and evaluation of sulfamide-type indolizidines as glycosidase inhibitors

21 January 2018

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Synthesis of thiohydantoin-castanospermine glycomimetics as glycosidase inhibitors.

21 January 2018

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Chaperone activity of bicyclic nojirimycin analogues for Gaucher mutations in comparison with N-(n-nonyl)deoxynojirimycin

21 January 2018

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6-Amino-6-deoxy-5,6-di-N-(N'-octyliminomethylidene)nojirimycin: synthesis, biological evaluation, and crystal structure in complex with acid beta-glucosidase

21 January 2018

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

21 January 2018

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A Fluorescent sp2-iminosugar with pharmacological chaperone activity for gaucher disease: synthesis and intracellular distribution studies

21 January 2018

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

21 January 2018

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

21 January 2018

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Molecular basis for beta-glucosidase inhibition by ring-modified calystegine analogues

21 January 2018

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Bicyclic derivatives of L-idonojirimycin as pharmacological chaperones for neuronopathic forms of Gaucher disease.

21 January 2018

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

21 January 2018

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Ubiquinone (coenzyme q10) and mitochondria in oxidative stress of parkinson's disease

21 January 2018

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Treatment effect of coenzyme Q(10) and an antioxidant cocktail in fibroblasts of patients with Sanfilippo disease

21 January 2018

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Promising results of the chaperone effect caused by imino sugars and aminocyclitol derivatives on mutant glucocerebrosidases causing Gaucher disease

21 January 2018

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Polyhydroxylated bicyclic isoureas and guanidines are potent glucocerebrosidase inhibitors and nanomolar enzyme activity enhancers in Gaucher cells.

21 January 2018

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The myo-1,2-Diaminocyclitol Scaffold Defines Potent Glucocerebrosidase Activators and Promising Pharmacological Chaperones for Gaucher Disease.

21 January 2018

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Selective chaperone effect of aminocyclitol derivatives on G202R and other mutant glucocerebrosidases causing Gaucher disease.

21 January 2018

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Galactonojirimycin derivatives restore mutant human beta-galactosidase activities expressed in fibroblasts from enzyme-deficient knockout mouse

21 January 2018

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The potential action of galactose as a "chemical chaperone": increase of beta galactosidase activity in fibroblasts from an adult GM1-gangliosidosis patient

21 January 2018

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Synthesis of lipophilic 1-deoxygalactonojirimycin derivatives as D-galactosidase inhibitors

21 January 2018

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1-Deoxy-D-galactonojirimycins with dansyl capped N-substituents as β-galactosidase inhibitors and potential probes for GM1 gangliosidosis affected cell lines.

21 January 2018

1 reference

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DLHex-DGJ, a novel derivative of 1-deoxygalactonojirimycin with pharmacological chaperone activity in human G(M1)-gangliosidosis fibroblasts

21 January 2018

1 reference

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Fluorous iminoalditols: a new family of glycosidase inhibitors and pharmacological chaperones.

21 January 2018

1 reference

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Fluorous iminoalditols act as effective pharmacological chaperones against gene products from GLB₁ alleles causing GM1-gangliosidosis and Morquio B disease

21 January 2018

1 reference

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A fluorescent probe for GM1 gangliosidosis related β-galactosidase: N-(dansylamino)hexylaminocarbonylpentyl-1,5-dideoxy-1,5-imino-D-galactitol.

21 January 2018

1 reference

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Evaluation of N-nonyl-deoxygalactonojirimycin as a pharmacological chaperone for human GM1 gangliosidosis leads to identification of a feline model suitable for testing enzyme enhancement therapy

21 January 2018

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Synthesis of 1,5-dideoxy-1,5-iminoribitol C-glycosides through a nitrone-olefin cycloaddition domino strategy: identification of pharmacological chaperones of mutant human lysosomal β-galactosidase.

21 January 2018

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N-Alkyl-, 1-C-Alkyl-, and 5-C-Alkyl-1,5-dideoxy-1,5-imino-(L)-ribitols as Galactosidase Inhibitors

21 January 2018

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Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis

21 January 2018

1 reference

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Chemical chaperone therapy: chaperone effect on mutant enzyme and cellular pathophysiology in β-galactosidase deficiency.

21 January 2018

1 reference

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Therapeutic chaperone effect of N-octyl 4-epi-β-valienamine on murine G(M1)-gangliosidosis.

21 January 2018

1 reference

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Chemical chaperone therapy: luciferase assay for screening of β-galactosidase mutations

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Crystal Structure of Human -Galactosidase: STRUCTURAL BASIS OF GM1 GANGLIOSIDOSIS AND MORQUIO B DISEASES

21 January 2018

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A bicyclic 1-deoxygalactonojirimycin derivative as a novel pharmacological chaperone for GM1 gangliosidosis.

21 January 2018

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Structural basis of pharmacological chaperoning for human β-galactosidase.

21 January 2018

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Galactose stabilizes various missense mutants of alpha-galactosidase in Fabry disease

21 January 2018

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Active-site-specific chaperone therapy for Fabry disease. Yin and Yang of enzyme inhibitors

21 January 2018

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Effects of a chemical chaperone on genetic mutations in alpha-galactosidase A in Korean patients with Fabry disease

21 January 2018

1 reference

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The pharmacological chaperone 1-deoxygalactonojirimycin increases alpha-galactosidase A levels in Fabry patient cell lines

21 January 2018

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The pharmacological chaperone 1-deoxygalactonojirimycin reduces tissue globotriaosylceramide levels in a mouse model of Fabry disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

A pharmacogenetic approach to identify mutant forms of α-galactosidase A that respond to a pharmacological chaperone for Fabry disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

A Phase 2 study of migalastat hydrochloride in females with Fabry disease: selection of population, safety and pharmacodynamic effects.

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

α-Galactosidase aggregation is a determinant of pharmacological chaperone efficacy on Fabry disease mutants

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Co-administration with the pharmacological chaperone AT1001 increases recombinant human α-galactosidase A tissue uptake and improves substrate reduction in Fabry mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Coformulation of a Novel Human α-Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry Mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

2,5-Dideoxy-2,5-imino-d-altritol as a new class of pharmacological chaperone for Fabry disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Effects of pH and Iminosugar Pharmacological Chaperones on Lysosomal Glycosidase Structure and Stability

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

The Molecular Basis of Pharmacological Chaperoning in Human α-Galactosidase

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Looking for protein stabilizing drugs with thermal shift assay.

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Molecular basis of 1-deoxygalactonojirimycin arylthiourea binding to human α-galactosidase a: pharmacological chaperoning efficacy on Fabry disease mutants

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Improvement in cardiac function in the cardiac variant of Fabry's disease with galactose-infusion therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

From blood-brain barrier to blood-brain interface: new opportunities for CNS drug delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Enhancing biodistribution of therapeutic enzymes in vivo by modulating surface coating and concentration of ICAM-1-targeted nanocarriers

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

pH-Responsive Pharmacological Chaperones for Rescuing Mutant Glycosidases.

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Fluorescence-quenched substrates for live cell imaging of human glucocerebrosidase activity.

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Visualization of Active Glucocerebrosidase in Rodent Brain with High Spatial Resolution following In Situ Labeling with Fluorescent Activity Based Probes

21 January 2018

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https://api.crossref.org/works/10.1039%2FC6CC01564F

Mechanism-based inhibitors of glycosidases: design and applications.

21 January 2018

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A chaperone enhances blood α-glucosidase activity in Pompe disease patients treated with enzyme replacement therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

The pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

The pharmacological chaperone AT2220 increases recombinant human acid α-glucosidase uptake and glycogen reduction in a mouse model of Pompe disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Azasugar inhibitors as pharmacological chaperones for Krabbe disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Chaperone therapy for Krabbe disease: potential for late-onset GALC mutations.

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Design, synthesis, and biological evaluation of enantiomeric beta-N-acetylhexosaminidase inhibitors LABNAc and DABNAc as potential agents against Tay-Sachs and Sandhoff disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Pharmacological chaperones for human -N-acetylgalactosaminidase

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Protein misfolding as an underlying molecular defect in mucopolysaccharidosis III type C

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

Correction of lysosomal dysfunction as a therapeutic strategy for neurodegenerative diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1039%2FC6CC01564F

21 January 2018

Identifiers

DOI

10.1039/C6CC01564F

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27043200%20AND%20SRC:MED&resulttype=core&format=json

31 March 2020

1 reference