(Q45856196)

17 December 2017

title

Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice (English)

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17 December 2017

author name string

Watson G

1

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17 December 2017

Bastacky J

2

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17 December 2017

Belichenko P

3

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17 December 2017

Buddhikot M

4

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17 December 2017

Jungles S

5

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17 December 2017

Vellard M

6

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17 December 2017

Mobley WC

7

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17 December 2017

Kakkis E

8

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17 December 2017

publication date

1 June 2006

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17 December 2017

published in

Gene Therapy

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17 December 2017

volume

13

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17 December 2017

issue

11

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17 December 2017

page(s)

917-925

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17 December 2017

https://scigraph.springernature.com/pub.10.1038/sj.gt.3302735

exact match

0 references

cites work

Cord-blood transplants from unrelated donors in patients with Hurler's syndrome

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Enzyme-replacement therapy in mucopolysaccharidosis I.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase).

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Prevention of neuropathology in the mouse model of Hurler syndrome

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Lysosomal storage diseases

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Enzyme replacement in a canine model of Hurler syndrome

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Enzyme replacement therapy in feline mucopolysaccharidosis I.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Laronidase treatment of mucopolysaccharidosis I.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Successful induction of immune tolerance to enzyme replacement therapy in canine mucopolysaccharidosis I

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Marrow transplantation in genetic disease

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Brain transplantation of genetically modified bone marrow stromal cells corrects CNS pathology and cognitive function in MPS VII mice

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Gene therapy progress and prospects: gene therapy of lysosomal storage disorders

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Effective gene therapy for an inherited CNS disease in a large animal model

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Enhanced secretion and uptake of beta-glucuronidase improves adeno-associated viral-mediated gene therapy of mucopolysaccharidosis type VII mice

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

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/10.1038%2FSJ.GT.3302735

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Interendothelial junctional changes underlie the developmental 'tightening' of the blood-brain barrier.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Efficient transduction of green fluorescent protein in spinal cord neurons using adeno-associated virus vectors containing cell type-specific promoters

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

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/10.1038%2FSJ.GT.3302735

7 January 2021

Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Enzyme replacement therapy for murine mucopolysaccharidosis type VII

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Overexpression of the human lysosomal enzyme alpha-L-iduronidase in Chinese hamster ovary cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Adeno-associated virus vectors can be efficiently produced without helper virus

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302735

7 January 2021

Identifiers

DOI

10.1038/SJ.GT.3302735

1 reference

17 December 2017

Dimensions Publication ID

0 references

1 reference

17 December 2017