Gene transfer may be preventive but not curative for a lysosomal transport disorder. (Q39968431)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:18578013%20AND%20SRC:MED&resulttype=core&format=json

9 January 2020

title

Gene transfer may be preventive but not curative for a lysosomal transport disorder (English)

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9 January 2020

10

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9 January 2020

Corinne Antignac

8

1 reference

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9 January 2020

Eric J Kremer

9

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9 January 2020

author name string

Claire Hippert

1

1 reference

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9 January 2020

Grégor Dubois

2

1 reference

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9 January 2020

Carole Morin

3

1 reference

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9 January 2020

Olivier Disson

4

1 reference

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9 January 2020

Sandy Ibanes

5

1 reference

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9 January 2020

Chantal Jacquet

6

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9 January 2020

Reto Schwendener

7

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9 January 2020

publication date

24 June 2008

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9 January 2020

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9 January 2020

volume

16

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9 January 2020

issue

8

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9 January 2020

page(s)

1372-1381

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

9 January 2020

cites work

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7 January 2021

Lysosomal storage diseases

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Cystinosin, the protein defective in cystinosis, is a H(+)-driven lysosomal cystine transporter

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Functional characterization of wild-type and mutant human sialin

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A novel gene encoding an integral membrane protein is mutated in nephropathic cystinosis

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Molecular pathogenesis of cystinosis: effect of CTNS mutations on the transport activity and subcellular localization of cystinosin

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Cystinosis. Intracellular cystine depletion by aminothiols in vitro and in vivo

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Intralysosomal cystine accumulation in mice lacking cystinosin, the protein defective in cystinosis

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The Ocular Anomalies in a Cystinosis Animal Model Mimic Disease Pathogenesis

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Rapid Kupffer cell death after intravenous injection of adenovirus vectors

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"In vivo" depletion of macrophages by liposome-mediated "suicide".

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Late symptoms in infantile cystinosis

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7 January 2021

Noncirrhotic portal hypertension in association with juvenile nephropathic cystinosis: case presentation and review of the literature

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7 January 2021

The targeting of cystinosin to the lysosomal membrane requires a tyrosine-based signal and a novel sorting motif

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7 January 2021

Immunolocalization of cystinosin, the protein defective in cystinosis

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

Long-term in vivo transduction of neurons throughout the rat CNS using novel helper-dependent CAV-2 vectors.

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7 January 2021

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