Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer (Q43747170)

26 November 2017

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

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26 November 2017

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Daly TM

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Ohlemiller KK

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26 November 2017

Roberts MS

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26 November 2017

Vogler CA

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26 November 2017

Sands MS

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1 September 2001

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1291-1298

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https://scigraph.springernature.com/pub.10.1038/sj.gt.3301420

26 November 2017

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cites work

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

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

21 January 2018

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Hepatic storage of glycosaminoglycans in feline and canine models of mucopolysaccharidoses I, VI, and VII.

21 January 2018

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

21 January 2018

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Structure and function of the mannose 6-phosphate/insulinlike growth factor II receptors

21 January 2018

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Enzyme replacement therapy for murine mucopolysaccharidosis type VII

21 January 2018

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

21 January 2018

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Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation

21 January 2018

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

21 January 2018

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

21 January 2018

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Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer

21 January 2018

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Site-specific integration by adeno-associated virus

21 January 2018

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Recombinant adeno-associated virus for muscle directed gene therapy

21 January 2018

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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.

21 January 2018

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Correction of murine mucopolysaccharidosis VII by a human beta-glucuronidase transgene

21 January 2018

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

21 January 2018

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Novel tools for production and purification of recombinant adenoassociated virus vectors

21 January 2018

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Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

21 January 2018

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Targeted deletion of the cytosolic Cu/Zn-superoxide dismutase gene (Sod1) increases susceptibility to noise-induced hearing loss

21 January 2018

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Inborn errors of mucopolysaccharide metabolism

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3301420

7 January 2021

Enzyme replacement with recombinant beta-glucuronidase in the newborn mucopolysaccharidosis type VII mouse

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7 January 2021

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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https://api.crossref.org/works/10.1038%2FSJ.GT.3301420

7 January 2021

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

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7 January 2021

Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers

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https://api.crossref.org/works/10.1038%2FSJ.GT.3301420

7 January 2021

Abnormal immune function in vivo in a murine model of lysosomal storage disease

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https://api.crossref.org/works/10.1038%2FSJ.GT.3301420

7 January 2021

Identifiers

DOI

10.1038/SJ.GT.3301420

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Dimensions Publication ID

26 November 2017

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