(Q44963505)

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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scholarly article

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Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:15233947%20AND%20SRC:MED&resulttype=core&format=json

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)

1 reference

Europe PubMed Central

PubMed publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:15233947%20AND%20SRC:MED&resulttype=core&format=json

30 December 2019

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Judith M Ogilvie

2

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:15233947%20AND%20SRC:MED&resulttype=core&format=json

30 December 2019

6

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Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:15233947%20AND%20SRC:MED&resulttype=core&format=json

30 December 2019

author name string

Anne K Hennig

1

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Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:15233947%20AND%20SRC:MED&resulttype=core&format=json

30 December 2019

Kevin K Ohlemiller

3

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Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:15233947%20AND%20SRC:MED&resulttype=core&format=json

30 December 2019

Adrian M Timmers

4

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30 December 2019

William W Hauswirth

5

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30 December 2019

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1 July 2004

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Europe PubMed Central

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30 December 2019

Molecular Therapy

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:15233947%20AND%20SRC:MED&resulttype=core&format=json

30 December 2019

10

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30 December 2019

1

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30 December 2019

106-116

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Europe PubMed Central

PubMed publication ID

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30 December 2019

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

Inborn errors of mucopolysaccharide metabolism

1 reference

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7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

Feline immunodeficiency virus vectors. Gene transfer to mouse retina following intravitreal injection

1 reference

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7 January 2021

Phenotypic rescue after adeno-associated virus-mediated delivery of 4-sulfatase to the retinal pigment epithelium of feline mucopolysaccharidosis VI.

1 reference

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7 January 2021

Tests of the mouse visual system.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

Generation of activated sialoadhesin-positive microglia during retinal degeneration

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

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

1 reference

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7 January 2021

In vivo gene therapy in young and adult RPE65-/- dogs produces long-term visual improvement.

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy.

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7 January 2021

Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration

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7 January 2021

Molecular genetics and prospects for therapy of the inherited retinal dystrophies

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7 January 2021

On the genetics of retinitis pigmentosa and on mutation-independent approaches to therapeutic intervention

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7 January 2021

Intraocular gene transfer of ciliary neurotrophic factor prevents death and increases responsiveness of rod photoreceptors in the retinal degeneration slow mouse

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

Long-term protection of retinal structure but not function using RAAV.CNTF in animal models of retinitis pigmentosa

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.018

7 January 2021

Effects of adeno-associated virus-vectored ciliary neurotrophic factor on retinal structure and function in mice with a P216L rds/peripherin mutation

1 reference

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7 January 2021

Evaluation of recombinant adeno-associated virus as a gene transfer vector for the retina

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7 January 2021

Novel tools for production and purification of recombinant adenoassociated virus vectors

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

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Growth factors in combination, but not individually, rescue rd mouse photoreceptors in organ culture

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HISTOCHEMICAL DEMONSTRATION OF N-ACETYL-BETA-GLUCOSAMINIDASE EMPLOYING NAPHTHOL AS-BI N-ACETYL-BETA -GLUCOSAMINIDE AS SUBSTRATE

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Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis

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Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer

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7 January 2021

based on heuristic

inferred from DOI database lookup

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