New prospects for the treatment of lysosomal storage diseases.
about
Varied mechanisms underlie the free sialic acid storage disordersProgression of multiple behavioral deficits with various ages of onset in a murine model of Hurler syndrome.Chemical chaperones increase the cellular activity of N370S beta -glucosidase: a therapeutic strategy for Gaucher disease.Chaperone therapy for neuronopathic lysosomal diseases: competitive inhibitors as chemical chaperones for enhancement of mutant enzyme activitiesModest phenotypic improvements in ASA-deficient mice with only one UDP-galactose:ceramide-galactosyltransferase gene.Carboxyl-terminal truncations alter the activity of the human α-galactosidase AEndocytic trafficking of glycosphingolipids in sphingolipid storage diseasesA historical perspective of the glycosphingolipids and sphingolipidoses.Electrocardiographic and other cardiac anomalies in beta-glucuronidase-null mice corrected by nonablative neonatal marrow transplantationFarber disease: clinical presentation, pathogenesis and a new approach to treatment.Weekly enzyme replacement therapy may slow decline of renal function in patients with Fabry disease who are on long-term biweekly dosing.Direct multiplex assay of lysosomal enzymes in dried blood spots for newborn screening.New developments in treating glycosphingolipid storage diseases.Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood-brain barrierComparative analysis of brain lipids in mice, cats, and humans with Sandhoff diseaseA counterintuitive approach to treat enzyme deficiencies: use of enzyme inhibitors for restoring mutant enzyme activity.Efficient gene therapy-based method for the delivery of therapeutics to primate cortex.Glucocerebroside: an evolutionary advantage for patients with Gaucher disease and a new immunomodulatory agent.Genistin-rich soy isoflavone extract in substrate reduction therapy for Sanfilippo syndrome: An open-label, pilot study in 10 pediatric patients.Myelin lesions associated with lysosomal and peroxisomal disorders.SDS-resistant active and thermostable dimers are obtained from the dissociation of homotetrameric beta-glycosidase from hyperthermophilic Sulfolobus solfataricus in SDS. Stabilizing role of the A-C intermonomeric interface.Substrate reduction reduces gangliosides in postnatal cerebrum-brainstem and cerebellum in GM1 gangliosidosis mice.Gem-diamine 1-N-iminosugars as versatile glycomimetics: synthesis, biological activity and therapeutic potential.Characterization of a thermostable family 1 Glycosyl Hydrolase enzyme from Putranjiva roxburghii seeds.
P2860
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P2860
New prospects for the treatment of lysosomal storage diseases.
description
2002 nî lūn-bûn
@nan
2002 թուականի Յունուարին հրատարակուած գիտական յօդուած
@hyw
2002 թվականի հունվարին հրատարակված գիտական հոդված
@hy
2002年の論文
@ja
2002年論文
@yue
2002年論文
@zh-hant
2002年論文
@zh-hk
2002年論文
@zh-mo
2002年論文
@zh-tw
2002年论文
@wuu
name
New prospects for the treatment of lysosomal storage diseases.
@ast
New prospects for the treatment of lysosomal storage diseases.
@en
New prospects for the treatment of lysosomal storage diseases.
@nl
type
label
New prospects for the treatment of lysosomal storage diseases.
@ast
New prospects for the treatment of lysosomal storage diseases.
@en
New prospects for the treatment of lysosomal storage diseases.
@nl
prefLabel
New prospects for the treatment of lysosomal storage diseases.
@ast
New prospects for the treatment of lysosomal storage diseases.
@en
New prospects for the treatment of lysosomal storage diseases.
@nl
P1433
P1476
New prospects for the treatment of lysosomal storage diseases.
@en
P2093
Raphael Schiffmann
Roscoe O Brady
P304
P356
10.2165/00003495-200262050-00002
P577
2002-01-01T00:00:00Z
P6179
1053356257