AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice. (Q44963505)

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scientific article published in July 2004

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English	AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice.	scientific article published in July 2004

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30 December 2019

AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice (English)

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30 December 2019

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Judith M Ogilvie

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30 December 2019

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Anne K Hennig

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30 December 2019

Kevin K Ohlemiller

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Adrian M Timmers

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William W Hauswirth

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1 July 2004

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30 December 2019

Molecular Therapy

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106-116

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30 December 2019

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

1 reference

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7 January 2021

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

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

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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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Beta-glucuronidase mediated pathway essential for retinal pigment epithelial degradation of glycosaminoglycans. Disease expression and in vitro disease correction using retroviral mediated cDNA transfer

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Inborn errors of mucopolysaccharide metabolism

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

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Adenovirus-mediated gene transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice

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Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus

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

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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.

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

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Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII.

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Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors

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Phenotype correction in retinal pigment epithelium in murine mucopolysaccharidosis VII by adenovirus-mediated gene transfer

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Tests of the mouse visual system.

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Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice.

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Generation of activated sialoadhesin-positive microglia during retinal degeneration

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Adenovirus-mediated gene therapy for corneal clouding in mice with mucopolysaccharidosis type VII.

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Impurity of recombinant adeno-associated virus type 2 affects the transduction characteristics following subretinal injection in the rat.

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

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Intraocular gene delivery of ciliary neurotrophic factor results in significant loss of retinal function in normal mice and in the Prph2Rd2/Rd2 model of retinal degeneration

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