Enzyme replacement in murine mucopolysaccharidosis type VII: neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy.
about
Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I.Characteristics of compounds that cross the blood-brain barrierInvolvement of the Toll-like receptor 4 pathway and use of TNF-alpha antagonists for treatment of the mucopolysaccharidosesCase report of treatment experience with idursulfase beta (Hunterase) in an adolescent patient with MPS II.Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIBMouse models of neurological disorders: a view from the blood-brain barrier.Brain-directed gene therapy for lysosomal storage disease: going well beyond the blood- brain barrier.Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.A breach in the blood-brain barrier.New strategies for enzyme replacement therapy for lysosomal storage diseasesSystemic administration of tripeptidyl peptidase I in a mouse model of late infantile neuronal ceroid lipofuscinosis: effect of glycan modification.Active site mutant transgene confers tolerance to human beta-glucuronidase without affecting the phenotype of MPS VII mice.Enzyme replacement therapy for the mucopolysaccharide storage disorders.Production of MPS VII mouse (Gus(tm(hE540A x mE536A)Sly)) doubly tolerant to human and mouse beta-glucuronidaseElectrocardiographic and other cardiac anomalies in beta-glucuronidase-null mice corrected by nonablative neonatal marrow transplantationEpinephrine enhances lysosomal enzyme delivery across the blood brain barrier by up-regulation of the mannose 6-phosphate receptorChronic enzyme replacement therapy ameliorates neuropathology in alpha-mannosidosis micePharmacologic manipulation of lysosomal enzyme transport across the blood-brain barrier.Animal models for mucopolysaccharidosis disorders and their clinical relevance.Blood-brain barrier transport of therapeutics via receptor-mediationDevelopmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier.Enzyme replacement therapy for Farber disease: Proof-of-concept studies in cells and mice.Pathogenesis and treatment of spine disease in the mucopolysaccharidoses.High and prolonged sulfamidase secretion by the liver of MPS-IIIA mice following hydrodynamic tail vein delivery of antibiotic-free pFAR4 plasmid vector.Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer.Long-term normalization in the central nervous system, ocular manifestations, and skeletal deformities by a single systemic adenovirus injection into neonatal mice with mucopolysaccharidosis VII.Attenuation of ganglioside GM1 accumulation in the brain of GM1 gangliosidosis mice by neonatal intravenous gene transfer.Efficacy of enzyme replacement therapy in an aggravated mouse model of metachromatic leukodystrophy declines with age.Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII.
P2860
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P2860
Enzyme replacement in murine mucopolysaccharidosis type VII: neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy.
description
1999 nî lūn-bûn
@nan
1999年の論文
@ja
1999年学术文章
@wuu
1999年学术文章
@zh-cn
1999年学术文章
@zh-hans
1999年学术文章
@zh-my
1999年学术文章
@zh-sg
1999年學術文章
@yue
1999年學術文章
@zh
1999年學術文章
@zh-hant
name
Enzyme replacement in murine m ...... of enzyme replacement therapy.
@en
Enzyme replacement in murine m ...... of enzyme replacement therapy.
@nl
type
label
Enzyme replacement in murine m ...... of enzyme replacement therapy.
@en
Enzyme replacement in murine m ...... of enzyme replacement therapy.
@nl
prefLabel
Enzyme replacement in murine m ...... of enzyme replacement therapy.
@en
Enzyme replacement in murine m ...... of enzyme replacement therapy.
@nl
P2093
P2860
P1433
P1476
Enzyme replacement in murine m ...... of enzyme replacement therapy.
@en
P2093
Birkenmeier EH
P2860
P304
P356
10.1203/00006450-199906000-00010
P407
P577
1999-06-01T00:00:00Z
P6179
1064216753