(Q46936588)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:17955027%20AND%20SRC:MED&resulttype=core&format=json

9 January 2020

title

Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle (English)

1 reference

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9 January 2020

main subject

mucopolysaccharidosis VI

1 reference

9

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9 January 2020

author name string

Alessandra Tessitore

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:17955027%20AND%20SRC:MED&resulttype=core&format=json

9 January 2020

Armida Faella

2

1 reference

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9 January 2020

Thomas O'Malley

3

1 reference

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9 January 2020

Gabriella Cotugno

4

1 reference

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9 January 2020

Monica Doria

5

1 reference

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9 January 2020

Tetsuo Kunieda

6

1 reference

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9 January 2020

Giuseppe Matarese

7

1 reference

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9 January 2020

Mark Haskins

8

1 reference

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9 January 2020

publication date

23 October 2007

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9 January 2020

1 reference

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9 January 2020

volume

16

1 reference

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9 January 2020

issue

1

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9 January 2020

page(s)

30-37

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The status of hematopoietic stem cell transplantation in lysosomal storage disease

9 January 2020

cites work

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7 January 2021

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7 January 2021

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7 January 2021

Two mutations within a feline mucopolysaccharidosis type VI colony cause three different clinical phenotypes

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7 January 2021

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

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7 January 2021

E-box sites and a proximal regulatory region of the muscle creatine kinase gene differentially regulate expression in diverse skeletal muscles and cardiac muscle of transgenic mice

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7 January 2021

Immune response to enzyme replacement therapy: 4-sulfatase epitope reactivity of plasma antibodies from MPS VI cats

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7 January 2021

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7 January 2021

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

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7 January 2021

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

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7 January 2021

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7 January 2021

Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

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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 correction of murine lipoprotein lipase deficiency with AAV1-mediated gene transfer of the naturally occurring LPL(S447X) beneficial mutation

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7 January 2021

Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutation

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7 January 2021

Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.

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7 January 2021

Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer

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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

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7 January 2021

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7 January 2021

Bone marrow transplantation in newborn rats with mucopolysaccharidosis type VI: biochemical, pathological, and clinical findings

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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

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7 January 2021

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7 January 2021

Overexpression of N-acetylgalactosamine-4-sulphatase induces a multiple sulphatase deficiency in mucopolysaccharidosis-type-VI fibroblasts

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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

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7 January 2021

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7 January 2021

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7 January 2021