Identification and characterization of ambroxol as an enzyme enhancement agent for Gaucher disease - Wikidata - awgldk - TriplyDB

Identification and characterization of ambroxol as an enzyme enhancement agent for Gaucher disease

Identification and characterization of ambroxol as an enzyme enhancement agent for Gaucher disease

http://www.wikidata.org/entity/Q33478327

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Glucocerebrosidase is shaking up the synucleinopathies An open-label Phase I/II clinical trial of pyrimethamine for the treatment of patients affected with chronic GM2 gangliosidosis (Tay-Sachs or Sandhoff variants)Recent developments in the use of differential scanning fluorometry in protein and small molecule discovery and characterization Emerging novel concept of chaperone therapies for protein misfolding diseases Cyclodextrin-mediated crystallization of acid β-glucosidase in complex with amphiphilic bicyclic nojirimycin analogues Binding of 3,4,5,6-Tetrahydroxyazepanes to the Acid-β-glucosidase Active Site: Implications for Pharmacological Chaperone Design for Gaucher Disease Converging roles of ion channels, calcium, metabolic stress, and activity pattern of Substantia nigra dopaminergic neurons in health and Parkinson's disease The relationship between glucocerebrosidase mutations and Parkinson disease Developing therapeutic approaches for metachromatic leukodystrophy Ambroxol improves lysosomal biochemistry in glucocerebrosidase mutation-linked Parkinson disease cells Characterization of the biosynthesis, processing and kinetic mechanism of action of the enzyme deficient in mucopolysaccharidosis IIIC Novel patient cell-based HTS assay for identification of small molecules for a lysosomal storage disease A rapid and sensitive method for measuring N-acetylglucosaminidase activity in cultured cells A Review of Gaucher Disease Pathophysiology, Clinical Presentation and Treatments 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.Fluorous iminoalditols: a new family of glycosidase inhibitors and pharmacological chaperones.The development and use of small molecule inhibitors of glycosphingolipid metabolism for lysosomal storage diseases.Gaucher iPSC-derived macrophages produce elevated levels of inflammatory mediators and serve as a new platform for therapeutic development.Development of targeted therapies for Parkinson's disease and related synucleinopathies A high-throughput screening assay using Krabbe disease patient cells.Expression of human Gaucher disease gene GBA generates neurodevelopmental defects and ER stress in Drosophila eye Candidate molecules for chemical chaperone therapy of GM1-gangliosidosis.Identification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders.Pharmacological chaperones facilitate the post-ER transport of recombinant N370S mutant β-glucocerebrosidase in plant cells: evidence that N370S is a folding mutant.Endoplasmic Reticulum-Targeted Subunit Toxins Provide a New Approach to Rescue Misfolded Mutant Proteins and Revert Cell Models of Genetic Diseases.[More than expectorant: new scientific data on ambroxol in the context of the treatment of bronchopulmonary diseases].Ambroxol chaperone therapy for neuronopathic Gaucher disease: A pilot study.β-Glucosidase 2 (GBA2) activity and imino sugar pharmacology.Parkinson disease-linked GBA mutation effects reversed by molecular chaperones in human cell and fly models.Review of the safety and efficacy of imiglucerase treatment of Gaucher disease.Ambroxol effects in glucocerebrosidase and α-synuclein transgenic mice.A molecular analysis of the GBA gene in Caucasian South Africans with Parkinson's disease.Progress and potential of non-inhibitory small molecule chaperones for the treatment of Gaucher disease and its implications for Parkinson disease.Emerging drugs for lysosomal storage diseases.Pharmacological small molecules for the treatment of lysosomal storage disorders.Pharmacological chaperone therapy for Gaucher disease: a patent review.A Guided Tour of the Structural Biology of Gaucher Disease: Acid-β-Glucosidase and Saposin C.Gaucher disease paradigm: from ERAD to comorbidity.Treatment options for lysosomal storage disorders: developing insights.Glucocerebrosidase inhibitors for the treatment of Gaucher disease.

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Identification and characterization of ambroxol as an enzyme enhancement agent for Gaucher disease

http://www.wikidata.org/entity/Q33478327

description

2009 nî lūn-bûn

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2009 թուականի Յուլիսին հրատարակուած գիտական յօդուած

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2009 թվականի հուլիսին հրատարակված գիտական հոդված

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2009年の論文

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2009年論文

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2009年論文

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2009年論文

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2009年論文

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2009年論文

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2009年论文

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Identification and characteriz ...... ment agent for Gaucher disease

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Identification and characteriz ...... ment agent for Gaucher disease

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Identification and characteriz ...... ment agent for Gaucher disease

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Identification and characteriz ...... ment agent for Gaucher disease

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Identification and characteriz ...... ment agent for Gaucher disease

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Identification and characteriz ...... ment agent for Gaucher disease

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JBC.M109.012393

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Journal of Biological Chemistry

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Identification and characteriz ...... ment agent for Gaucher disease

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P2093

Brigitte A Rigat

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Deepangi Pandit

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Don J Mahuran

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Gregory J Kornhaber

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Joe T R Clarke

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Justin D Buttner

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Liangiie Tang

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Maria Fuller

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Michael B Tropak

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Yoshitomo Hamuro

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P2860

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

Determination of amide hydrogen exchange by mass spectrometry: a new tool for protein structure elucidation

A simple method for the isolation and purification of total lipides from animal tissues

Structure of acid beta-glucosidase with pharmacological chaperone provides insight into Gaucher disease

Signal integration in the endoplasmic reticulum unfolded protein response

The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: a position statement

Ambroxol in the 21st century: pharmacological and clinical update

Python: a programming language for software integration and development

Accuracy and precision of a new H/D exchange- and mass spectrometry-based technique for measuring the thermodynamic properties of protein-peptide complexes.

Rapid analysis of protein structure and dynamics by hydrogen/deuterium exchange mass spectrometry.

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23502-23516

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scholarly article

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10.1074/JBC.M109.012393

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35

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284

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Gustavo Maegawa

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2009-07-03T00:00:00Z

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19578116

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