Enzyme-replacement therapy for metabolic storage disorders.
about
Friends or Foes: Matrix Metalloproteinases and Their Multifaceted Roles in Neurodegenerative DiseasesKinetic and structural evidences on human prolidase pathological mutants suggest strategies for enzyme functional rescueCandidate molecules for chemical chaperone therapy of GM1-gangliosidosis.Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice.Carboxyl-terminal truncations alter the activity of the human α-galactosidase AEffects of cyclodextrins on GM1-gangliosides in fibroblasts from GM1-gangliosidosis patients.Delineating the role of alterations in lipid metabolism to the pathogenesis of inherited skeletal and cardiac muscle disorders: Thematic Review Series: Genetics of Human Lipid Diseases.Glycemic control and chronic dosing of rhesus monkeys with a fusion protein of iduronidase and a monoclonal antibody against the human insulin receptor.A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA.Novel candidate disease for gene therapy: metachromatic leukodystrophy.AGT-181: expression in CHO cells and pharmacokinetics, safety, and plasma iduronidase enzyme activity in Rhesus monkeys."Atypical" atypical parkinsonism: new genetic conditions presenting with features of progressive supranuclear palsy, corticobasal degeneration, or multiple system atrophy-a diagnostic guide.Targeted endothelial nanomedicine for common acute pathological conditions.Molecular logistics using cytocleavable polyrotaxanes for the reactivation of enzymes delivered in living cells.Glycosaminoglycan levels and structure in a mucopolysaccharidosis IIIA mice and the effect of a highly secreted sulfamidase engineered to cross the blood-brain barrier.Enzyme replacement improves ataxic gait and central nervous system histopathology in a mouse model of metachromatic leukodystrophy.Enzyme replacement for GM1-gangliosidosis: Uptake, lysosomal activation, and cellular disease correction using a novel β-galactosidase:RTB lectin fusion.Replacement of the C6ORF66 assembly factor (NDUFAF4) restores complex I activity in patient cells.Selective plasma pharmacokinetics and brain uptake in the mouse of enzyme fusion proteins derived from species-specific receptor-targeted antibodies.Receiving enzyme replacement therapy for a lysosomal storage disorder: a preliminary exploration of the experiences of young patients and their families.Substrate specificity and reaction mechanism of human prolidase.Chemical chaperone therapy: chaperone effect on mutant enzyme and cellular pathophysiology in β-galactosidase deficiency.Fabry disease
P2860
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P2860
Enzyme-replacement therapy for metabolic storage disorders.
description
2004 nî lūn-bûn
@nan
2004 թուականի Դեկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2004 թվականի դեկտեմբերին հրատարակված գիտական հոդված
@hy
2004年の論文
@ja
2004年論文
@yue
2004年論文
@zh-hant
2004年論文
@zh-hk
2004年論文
@zh-mo
2004年論文
@zh-tw
2004年论文
@wuu
name
Enzyme-replacement therapy for metabolic storage disorders.
@ast
Enzyme-replacement therapy for metabolic storage disorders.
@en
type
label
Enzyme-replacement therapy for metabolic storage disorders.
@ast
Enzyme-replacement therapy for metabolic storage disorders.
@en
prefLabel
Enzyme-replacement therapy for metabolic storage disorders.
@ast
Enzyme-replacement therapy for metabolic storage disorders.
@en
P1433
P1476
Enzyme-replacement therapy for metabolic storage disorders.
@en
P2093
Raphael Schiffmann
Roscoe O Brady
P304
P356
10.1016/S1474-4422(04)00938-X
P577
2004-12-01T00:00:00Z