(Q45855941)

17 December 2017

title

Neonatal gene transfer using lentiviral vector for murine Pompe disease: long-term expression and glycogen reduction (English)

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17 December 2017

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S O Kyosen

1

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17 December 2017

S Iizuka

2

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17 December 2017

H Kobayashi

3

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17 December 2017

T Kimura

4

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17 December 2017

T Fukuda

5

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17 December 2017

J Shen

6

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17 December 2017

Y Shimada

7

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17 December 2017

H Ida

8

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17 December 2017

Y Eto

9

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17 December 2017

T Ohashi

10

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17 December 2017

publication date

24 December 2009

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17 December 2017

published in

Gene Therapy

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17 December 2017

volume

17

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17 December 2017

issue

4

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17 December 2017

page(s)

521-530

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17 December 2017

https://scigraph.springernature.com/pub.10.1038/gt.2009.160

exact match

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cites work

Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease).

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

Pompe disease in infants and children

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A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease

21 January 2018

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21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Pompe disease: a review of the current diagnosis and treatment recommendations in the era of enzyme replacement therapy

21 January 2018

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Efficacy of gene therapy for a prototypical lysosomal storage disease (GSD-II) is critically dependent on vector dose, transgene promoter, and the tissues targeted for vector transduction

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II.

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

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

21 January 2018

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Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter

21 January 2018

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Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors

21 January 2018

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Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors

21 January 2018

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Gene therapy of storage disorders by retroviral and lentiviral vectors

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

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

21 January 2018

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Correction of glycogenosis type 2 by muscle-specific lentiviral vector

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Lentiviral vectors with CMV or MHCII promoters administered in vivo: immune reactivity versus persistence of expression

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

Reduced alpha-Gal A enzyme activity in Fabry fibroblast cells and Fabry mice tissues induced by serum from antibody positive patients with Fabry disease

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Induction of tolerance to a recombinant human enzyme, acid alpha-glucosidase, in enzyme deficient knockout mice

21 January 2018

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Immunology of neonatal gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

Mucopolysaccharidosis I cats mount a cytotoxic T lymphocyte response after neonatal gene therapy that can be blocked with CTLA4-Ig.

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

Gene transfer in humans using a conditionally replicating lentiviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

Limited transgene immune response and long-term expression of human alpha-L-iduronidase in young adult mice with mucopolysaccharidosis type I by liver-directed gene therapy

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Lentiviral-mediated gene therapy for murine mucopolysaccharidosis type IIIA.

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/progenitor cells

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Partial phenotypic correction and immune tolerance induction to enzyme replacement therapy after hematopoietic stem cell gene transfer of alpha-glucosidase in Pompe disease

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Autophagy and lysosomes in Pompe disease

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Dysfunction of endocytic and autophagic pathways in a lysosomal storage disease.

21 January 2018

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Deconstructing Pompe disease by analyzing single muscle fibers: to see a world in a grain of sand...

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

When more is less: excess and deficiency of autophagy coexist in skeletal muscle in Pompe disease

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Autophagy and mistargeting of therapeutic enzyme in skeletal muscle in Pompe disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

Impaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expression

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

Replacing acid alpha-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

Long-term efficacy after [E1-, polymerase-] adenovirus-mediated transfer of human acid-alpha-glucosidase gene into glycogen storage disease type II knockout mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

ABO-incompatible heart transplantation in infants

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Early detection of Pompe disease by newborn screening is feasible: results from the Taiwan screening program.

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Induction of immune tolerance by neonatal intravenous injection of human factor VIII in murine hemophilia A.

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

21 January 2018

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The use of lentiviral vectors in gene therapy of leukemia: combinatorial gene delivery of immunomodulators into leukemia cells by state-of-the-art vectors

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

Targeted disruption of the acid alpha-glucosidase gene in mice causes an illness with critical features of both infantile and adult human glycogen storage disease type II

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.160

A third-generation lentivirus vector with a conditional packaging system

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

7 January 2021

inferred from DOI database lookup

Immune tolerance induction to enzyme-replacement therapy by co-administration of short-term, low-dose methotrexate in a murine Pompe disease model

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

7 January 2021

inferred from DOI database lookup

Effects of epigenetic modulation on reporter gene expression: implications for stem cell imaging.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.160

7 January 2021

inferred from DOI database lookup

Identifiers

DOI

10.1038/GT.2009.160

1 reference

17 December 2017

Dimensions Publication ID

1030322456

0 references

Fatcat ID

release_gk4xkfkbhrhlppphqce2x5ftoy

1 reference

Fatcat

https://api.fatcat.wiki/v0/release/gk4xkfkbhrhlppphqce2x5ftoy

24 November 2022

mapped directly with Wikidata item

PubMed ID

20033064

1 reference

17 December 2017