Clinical response to persistent, low-level beta-glucuronidase expression in the murine model of mucopolysaccharidosis type VII. (Q48269203)

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25 December 2019

title

Clinical response to persistent, low-level beta-glucuronidase expression in the murine model of mucopolysaccharidosis type VII (English)

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25 December 2019

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1

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25 December 2019

Mark S Sands

series ordinal

4

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25 December 2019

author name string

B Levy

series ordinal

2

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25 December 2019

C Vogler

series ordinal

3

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25 December 2019

publication date

16 February 2007

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Journal of Inherited Metabolic Disease

25 December 2019

published in

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volume

30

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25 December 2019

issue

2

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25 December 2019

page(s)

227-238

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https://scigraph.springernature.com/pub.10.1007/s10545-007-0483-4

25 December 2019

exact match

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Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors

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Syngeneic bone marrow transplantation reduces the hearing loss associated with murine mucopolysaccharidosis type VII.

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Enzyme replacement with recombinant beta-glucuronidase in murine mucopolysaccharidosis type VII: impact of therapy during the first six weeks of life on subsequent lysosomal storage, growth, and survival

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A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII

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Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

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Retinal function is improved in a murine model of a lysosomal storage disease following bone marrow transplantation

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Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer

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Low beta-glucuronidase enzyme activity and mutations in the human beta-glucuronidase gene in mild mucopolysaccharidosis type VII, pseudodeficiency and a heterozygote

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Enzyme replacement therapy for murine mucopolysaccharidosis type VII

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Increased life span and correction of metabolic defects in murine mucopolysaccharidosis type VII after syngeneic bone marrow transplantation.

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Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency

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Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts

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Human beta-glucuronidase: in vivo clearance and in vitro uptake by a glycoprotein recognition system on reticuloendothelial cells

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Delivery of a retroviral vector expressing human beta-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice

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Widespread correction of lysosomal storage following intrahepatic injection of a recombinant adeno-associated virus in the adult MPS VII mouse

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Early onset of lysosomal storage disease in a murine model of mucopolysaccharidosis type VII: undegraded substrate accumulates in many tissues in the fetus and very young MPS VII mouse

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Gene therapy for lysosomal storage diseases.

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12 December 2020

Nonablative neonatal marrow transplantation attenuates functional and physical defects of beta-glucuronidase deficiency.

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12 December 2020

Photoreceptor degeneration and altered distribution of interphotoreceptor matrix proteoglycans in the mucopolysaccharidosis VII mouse

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12 December 2020

Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice.

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12 December 2020

Prevalence of lysosomal storage disorders

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12 December 2020

Electrocardiographic and other cardiac anomalies in beta-glucuronidase-null mice corrected by nonablative neonatal marrow transplantation

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12 December 2020

Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function

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12 December 2020

Glycogen storage in multiple muscles of old GSD-II mice can be rapidly cleared after a single intravenous injection with a modified adenoviral vector expressing hGAA.

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12 December 2020

Enzyme replacement therapy improves reproductive performance in mucopolysaccharidosis type VII mice but does not prevent postnatal losses.

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12 December 2020

Novel tools for production and purification of recombinant adenoassociated virus vectors

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12 December 2020

Numerous transcriptional alterations in liver persist after short-term enzyme-replacement therapy in a murine model of mucopolysaccharidosis type VII.

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12 December 2020

Enzyme replacement in murine mucopolysaccharidosis type VII: neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy.

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12 December 2020

Inner ear pathology in the mucopolysaccharidosis VII mouse.

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12 December 2020

Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis

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12 December 2020

Phenotype correction in murine mucopolysaccharidosis type VII by transplantation of human amniotic epithelial cells after adenovirus-mediated gene transfer

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Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.

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12 December 2020

Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII.

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12 December 2020

Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates.

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Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer

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12 December 2020

Gene therapy for lysosomal storage diseases: the lessons and promise of animal models

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Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector

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12 December 2020

Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene

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12 December 2020

Abnormal osteoclast morphology and bone remodeling in a murine model of a lysosomal storage disease.

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12 December 2020

Inborn errors of mucopolysaccharide metabolism

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12 December 2020

Quantitative evaluation of liver-specific promoters from retroviral vectors after in vivo transduction of hepatocytes

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12 December 2020

Gene transfer of low levels of beta-glucuronidase corrects hepatic lysosomal storage in a large animal model of mucopolysaccharidosis VII.

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12 December 2020

Percutaneous intravenous injection in neonatal mice

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12 December 2020

Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

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12 December 2020

Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.

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12 December 2020

10.1007/S10545-007-0483-4

Identifiers

DOI

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:17308887%20AND%20SRC:MED&resulttype=core&format=json

25 December 2019

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