Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer (Q30498316)

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17 January 2020

title

Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer (English)

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17 January 2020

main subject

mucopolysaccharidosis VI

1 reference

18

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11

Meg M Sleeper

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17 January 2020

15

John Hopwood

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17 January 2020

author name string

Gabriella Cotugno

1

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17 January 2020

Patrizia Annunziata

2

1 reference

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17 January 2020

Alessandra Tessitore

3

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17 January 2020

Thomas O'Malley

4

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17 January 2020

Anita Capalbo

5

1 reference

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17 January 2020

Armida Faella

6

1 reference

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17 January 2020

Rosa Bartolomeo

7

1 reference

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17 January 2020

Patricia O'Donnell

8

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17 January 2020

Ping Wang

9

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17 January 2020

Fabio Russo

10

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17 January 2020

Van W Knox

12

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17 January 2020

Steven Fernandez

13

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17 January 2020

Leah Levanduski

14

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17 January 2020

Elvira De Leonibus

16

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17 January 2020

Mark Haskins

17

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17 January 2020

publication date

30 November 2010

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17 January 2020

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17 January 2020

volume

19

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17 January 2020

page(s)

461-469

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17 January 2020

issue

3

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The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques

17 January 2020

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1 reference

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Targeted disruption of the arylsulfatase B gene results in mice resembling the phenotype of mucopolysaccharidosis VI

28 July 2018

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26 November 2018

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26 November 2018

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26 November 2018

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26 November 2018

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26 November 2018

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26 November 2018

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26 November 2018

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26 November 2018

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26 November 2018

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Optimization of the human factor VIII complementary DNA expression plasmid for gene therapy of hemophilia A.

26 November 2018

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Feline mucopolysaccharidosis type VI. Characterization of recombinant N-acetylgalactosamine 4-sulfatase and identification of a mutation causing the disease

26 November 2018

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12 December 2020

Cardiovascular changes in children with mucopolysaccharide storage diseases and related disorders--clinical and echocardiographic findings in 64 patients

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12 December 2020

Histomorphometric analysis of the tibial growth plate in a feline model of mucopolysaccharidosis type VI

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12 December 2020

Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, o

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12 December 2020

Newborn screening for lysosomal storage disorders

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12 December 2020

Expensive drugs for rare disorders: to treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI

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12 December 2020

Enzyme replacement therapy for mucopolysaccharidosis VI from 8 weeks of age--a sibling control study

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12 December 2020

Differential assay of arylsulfatase A and B activities: A sensitive method for cultured human cells

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12 December 2020

Growth plate pathology in feline mucopolysaccharidosis VI

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12 December 2020

Effect of enzyme replacement therapy on bone formation in a feline model of mucopolysaccharidosis type VI

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12 December 2020

Identifiers

DOI

10.1038/MT.2010.257

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21119624%20AND%20SRC:MED&resulttype=core&format=json

17 January 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21119624%20AND%20SRC:MED&resulttype=core&format=json

17 January 2020

1 reference