Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis.
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Update on treatment of lysosomal storage diseasesHot spots in prion protein for pathogenic conversionThe iminosugar isofagomine increases the activity of N370S mutant acid beta-glucosidase in Gaucher fibroblasts by several mechanismsSialic acids in the brain: gangliosides and polysialic acid in nervous system development, stability, disease, and regenerationEmerging novel concept of chaperone therapies for protein misfolding diseasesPartial restoration of mutant enzyme homeostasis in three distinct lysosomal storage disease cell lines by altering calcium homeostasisA rapid and sensitive method for measuring N-acetylglucosaminidase activity in cultured cellsVariety of antiprion compounds discovered through an in silico screen based on cellular-form prion protein structure: Correlation between antiprion activity and binding affinity.Current themes in molecular pediatrics: molecular medicine and its applications.Pyrimethamine as a potential pharmacological chaperone for late-onset forms of GM2 gangliosidosis.High-throughput screening for human lysosomal beta-N-Acetyl hexosaminidase inhibitors acting as pharmacological chaperones.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 pharmacological chaperone 1-deoxygalactonojirimycin reduces tissue globotriaosylceramide levels in a mouse model of Fabry disease.High throughput screening for inhibitors of alpha-galactosidase.Bicyclic (galacto)nojirimycin analogues as glycosidase inhibitors: effect of structural modifications in their pharmacological chaperone potential towards β-glucocerebrosidase.Amino acid residues critical for endoplasmic reticulum export and trafficking of platelet-activating factor receptorFabry disease - current treatment and new drug development.Restoration of testis function in hypogonadotropic hypogonadal mice harboring a misfolded GnRHR mutant by pharmacoperone drug therapy.Increased globotriaosylceramide levels in a transgenic mouse expressing human alpha1,4-galactosyltransferase and a mouse model for treating Fabry disease.Chaperone therapy for neuronopathic lysosomal diseases: competitive inhibitors as chemical chaperones for enhancement of mutant enzyme activitiesCandidate molecules for chemical chaperone therapy of GM1-gangliosidosis.Identification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders.The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseasesPharmacological chaperone therapy for Fabry diseaseStorage solutions: treating lysosomal disorders of the brain.Current and emerging therapies for the lysosomal storage disorders.Protein quality control: the who's who, the where's and therapeutic escapes.Ambroxol chaperone therapy for neuronopathic Gaucher disease: A pilot study.Pharmacotherapeutic strategies using small molecules for the treatment of glycolipid lysosomal storage disorders.Chemical and pharmacological chaperones as new therapeutic agents.Isofagomine- and 2,5-anhydro-2,5-imino-D-glucitol-based glucocerebrosidase pharmacological chaperones for Gaucher disease intervention.Active-site-specific chaperone therapy for Fabry disease. Yin and Yang of enzyme inhibitors.Dependence of reversibility and progression of mouse neuronopathic Gaucher disease on acid beta-glucosidase residual activity levels.A counterintuitive approach to treat enzyme deficiencies: use of enzyme inhibitors for restoring mutant enzyme activity.Screening for pharmacological chaperones in Fabry diseaseChemical chaperone and inhibitor discovery: potential treatments for protein conformational diseases.Chemical chaperones protect from effects of apoptosis-inducing mutation in carbonic anhydrase IV identified in retinitis pigmentosa 17.Progranulin Recruits HSP70 to β-Glucocerebrosidase and Is Therapeutic Against Gaucher Disease.Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics.
P2860
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P2860
Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis.
description
2003 nî lūn-bûn
@nan
2003 թուականի Դեկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2003 թվականի դեկտեմբերին հրատարակված գիտական հոդված
@hy
2003年の論文
@ja
2003年学术文章
@wuu
2003年学术文章
@zh-cn
2003年学术文章
@zh-hans
2003年学术文章
@zh-my
2003年学术文章
@zh-sg
2003年學術文章
@yue
name
Chemical chaperone therapy for brain pathology in G
@nl
Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis.
@ast
Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis.
@en
type
label
Chemical chaperone therapy for brain pathology in G
@nl
Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis.
@ast
Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis.
@en
prefLabel
Chemical chaperone therapy for brain pathology in G
@nl
Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis.
@ast
Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis.
@en
P2093
P2860
P356
P1476
Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis.
@en
P2093
Akihiro Oshima
Akira Noguchi
Eiji Nanba
Hiroshi Watanabe
Hiroyuki Iwasaki
Junichiro Matsuda
Katsumi Higaki
Kazuhiro Takimoto
Kousaku Ohno
Lika Tominaga
P2860
P304
15912-15917
P356
10.1073/PNAS.2536657100
P407
P577
2003-12-15T00:00:00Z