(Q45856355)

17 December 2017

title

Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery (English)

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17 December 2017

Maria Pia Cosma

8

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Alberto Auricchio

object named as

Alberto Auricchio

6

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Andrea Ballabio

object named as

Andrea Ballabio

7

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author name string

Monica Cardone

1

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17 December 2017

Vinicia Assunta Polito

2

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17 December 2017

Stefano Pepe

3

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17 December 2017

Linda Mann

4

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17 December 2017

Alessandra D'Azzo

5

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17 December 2017

publication date

27 February 2006

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17 December 2017

published in

Human Molecular Genetics

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17 December 2017

volume

15

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17 December 2017

issue

7

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17 December 2017

page(s)

1225-1236

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17 December 2017

The mucopolysaccharidoses: a heterogeneous group of disorders with variable pediatric presentations

cites work

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDL038

The multiple sulfatase deficiency gene encodes an essential and limiting factor for the activity of sulfatases

21 January 2018

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Multiple sulfatase deficiency is caused by mutations in the gene encoding the human C(alpha)-formylglycine generating enzyme

21 January 2018

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Molecular basis of mucopolysaccharidosis type II: mutations in the iduronate-2-sulphatase gene

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDL038

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

21 January 2018

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Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

21 January 2018

1 reference

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Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDL038

Sustained correction of bleeding disorder in hemophilia B mice by gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDL038

Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDL038

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDL038

Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDL038

Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDL038

Clinical and morphologic features of mucopolysaccharidosis type II in a dog: naturally occurring model of Hunter syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDL038

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDL038

Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDL038

The mannose 6-phosphate receptor and the biogenesis of lysosomes

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDL038

21 January 2018

Identifiers

DOI

10.1093/HMG/DDL038

1 reference

17 December 2017

PubMed ID

16505002

1 reference

17 December 2017