High throughput screening for small molecule therapy for Gaucher disease using patient tissue as the source of mutant glucocerebrosidase
about
Glucocerebrosidase is shaking up the synucleinopathiesAdvancing Biological Understanding and Therapeutics Discovery with Small-Molecule ProbesEmerging novel concept of chaperone therapies for protein misfolding diseasesTool compounds robustly increase turnover of an artificial substrate by glucocerebrosidase in human brain lysatesA high-throughput sphingomyelinase assay using natural substrate.Macrophage models of Gaucher disease for evaluating disease pathogenesis and candidate drugs.Development of targeted therapies for Parkinson's disease and related synucleinopathiesDiscovery, structure-activity relationship, and biological evaluation of noninhibitory small molecule chaperones of glucocerebrosidasePhenylalanine hydroxylase misfolding and pharmacological chaperones.New macrophage models of Gaucher disease offer new tools for drug developmentAtp13a2-deficient mice exhibit neuronal ceroid lipofuscinosis, limited α-synuclein accumulation and age-dependent sensorimotor deficits.A New Glucocerebrosidase Chaperone Reduces α-Synuclein and Glycolipid Levels in iPSC-Derived Dopaminergic Neurons from Patients with Gaucher Disease and Parkinsonism.Chaperoning glucocerebrosidase: a therapeutic strategy for both Gaucher disease and ParkinsonismDisease models for the development of therapies for lysosomal storage diseases.Progress and potential of non-inhibitory small molecule chaperones for the treatment of Gaucher disease and its implications for Parkinson disease.Gaucher disease paradigm: from ERAD to comorbidity.Glucocerebrosidase inhibitors for the treatment of Gaucher disease.Pharmacological chaperones for enzyme enhancement therapy in genetic diseases.Emerging therapeutic targets for Gaucher disease.Gaucher disease: Progress and ongoing challenges.The Complicated Relationship between Gaucher Disease and Parkinsonism: Insights from a Rare Disease.Drug repositioning can accelerate discovery of pharmacological chaperones.Ambroxol as a pharmacological chaperone for mutant glucocerebrosidase.Pharmacoperones as Novel Therapeutics for Diverse Protein Conformational Diseases.Successful desensitization to imiglucerase of an adult patient diagnosed with type I Gaucher disease.Dysregulation of the autophagic-lysosomal pathway in Gaucher and Parkinson's disease.
P2860
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P2860
High throughput screening for small molecule therapy for Gaucher disease using patient tissue as the source of mutant glucocerebrosidase
description
2012 nî lūn-bûn
@nan
2012 թուականին հրատարակուած գիտական յօդուած
@hyw
2012 թվականին հրատարակված գիտական հոդված
@hy
2012年の論文
@ja
2012年論文
@yue
2012年論文
@zh-hant
2012年論文
@zh-hk
2012年論文
@zh-mo
2012年論文
@zh-tw
2012年论文
@wuu
name
High throughput screening for ...... e of mutant glucocerebrosidase
@ast
High throughput screening for ...... e of mutant glucocerebrosidase
@en
High throughput screening for ...... e of mutant glucocerebrosidase
@nl
type
label
High throughput screening for ...... e of mutant glucocerebrosidase
@ast
High throughput screening for ...... e of mutant glucocerebrosidase
@en
High throughput screening for ...... e of mutant glucocerebrosidase
@nl
prefLabel
High throughput screening for ...... e of mutant glucocerebrosidase
@ast
High throughput screening for ...... e of mutant glucocerebrosidase
@en
High throughput screening for ...... e of mutant glucocerebrosidase
@nl
P2093
P2860
P50
P1433
P1476
High throughput screening for ...... e of mutant glucocerebrosidase
@en
P2093
Ehud Goldin
Ellen Sidransky
Jae Hyuk Choi
Juan Marugan
Omid Motabar
P2860
P304
P356
10.1371/JOURNAL.PONE.0029861
P407
P577
2012-01-01T00:00:00Z