Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation.
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Clinical course of sly syndrome (mucopolysaccharidosis type VII)Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I.Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIBBrain-directed gene therapy for lysosomal storage disease: going well beyond the blood- brain barrier.Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors.Bone marrow transplantation restores epidermal basement membrane protein expression and rescues epidermolysis bullosa model mice.Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.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.Transgene produces massive overexpression of human beta -glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors.Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?Production of MPS VII mouse (Gus(tm(hE540A x mE536A)Sly)) doubly tolerant to human and mouse beta-glucuronidaseCombination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse.Epinephrine enhances lysosomal enzyme delivery across the blood brain barrier by up-regulation of the mannose 6-phosphate receptorCNS-directed gene therapy for lysosomal storage diseasesBiochemical evidence for superior correction of neuronal storage by chemically modified enzyme in murine mucopolysaccharidosis VII.Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?Correction of murine galactosialidosis by bone marrow-derived macrophages overexpressing human protective protein/cathepsin A under control of the colony-stimulating factor-1 receptor promoter.Animal models for mucopolysaccharidosis disorders and their clinical relevance.Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII miceEnzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function.Bone marrow transplantation prolongs life span and ameliorates neurologic manifestations in Sandhoff disease mice.Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier.Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease.Bone marrow transplantation in epidermolysis bullosa.Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.Randomized controlled trial of Hepatitis B virus vaccine in HIV-1-infected patients comparing two different doses.Promising CNS-directed enzyme replacement therapy for lysosomal storage diseases.Numerous transcriptional alterations in liver persist after short-term enzyme-replacement therapy in a murine model of mucopolysaccharidosis type VII.Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer.Feline immunodeficiency virus vectors. Gene transfer to mouse retina following intravitreal injection.Low-calcium diet in hypercalciuric enuretic children restores AQP2 excretion and improves clinical symptoms.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.Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.Mucopolysaccharidosis VII in a cat.Enzyme-replacement therapy from birth delays the development of behavior and learning problems in mucopolysaccharidosis type IIIA mice.Enzyme replacement in murine mucopolysaccharidosis type VII: neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy.Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII.A novel model of murine mucopolysaccharidosis type VII due to an intracisternal a particle element transposition into the beta-glucuronidase gene: clinical and pathologic findings.
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Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation.
description
article científic
@ca
article scientifique
@fr
articolo scientifico
@it
artigo científico
@pt
bilimsel makale
@tr
scientific article published on April 1997
@en
vedecký článok
@sk
vetenskaplig artikel
@sv
videnskabelig artikel
@da
vědecký článek
@cs
name
Murine mucopolysaccharidosis t ...... y bone marrow transplantation.
@en
Murine mucopolysaccharidosis t ...... y bone marrow transplantation.
@nl
type
label
Murine mucopolysaccharidosis t ...... y bone marrow transplantation.
@en
Murine mucopolysaccharidosis t ...... y bone marrow transplantation.
@nl
prefLabel
Murine mucopolysaccharidosis t ...... y bone marrow transplantation.
@en
Murine mucopolysaccharidosis t ...... y bone marrow transplantation.
@nl
P2093
P2860
P356
P1476
Murine mucopolysaccharidosis t ...... y bone marrow transplantation.
@en
P2093
E H Birkenmeier
P2860
P304
P356
10.1172/JCI119322
P407
P577
1997-04-01T00:00:00Z