Widespread biochemical correction of murine mucopolysaccharidosis type VII pathology by liver hydrodynamic plasmid delivery (Q44791009)

6 December 2017

Widespread biochemical correction of murine mucopolysaccharidosis type VII pathology by liver hydrodynamic plasmid delivery (English)

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6 December 2017

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M Richard

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6 December 2017

A Arfi

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6 December 2017

J Seguin

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6 December 2017

C Gandolphe

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6 December 2017

D Scherman

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6 December 2017

9 April 2009

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6 December 2017

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6 December 2017

16

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746-756

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6

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https://scigraph.springernature.com/pub.10.1038/gt.2009.36

6 December 2017

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Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency

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Murine mucopolysaccharidosis VIL: impact of therapies on the phenotype, clinical course, and pathology in a model of a lysosomal storage disease

21 January 2018

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Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus

21 January 2018

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Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts

21 January 2018

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Enzyme replacement therapy for murine mucopolysaccharidosis type VII

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Gene transfer of low levels of beta-glucuronidase corrects hepatic lysosomal storage in a large animal model of mucopolysaccharidosis VII.

21 January 2018

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Clinical response to persistent, low-level beta-glucuronidase expression in the murine model of mucopolysaccharidosis type VII.

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Gene therapy for lysosomal storage diseases.

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

21 January 2018

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High-efficiency gene transfer into skeletal muscle mediated by electric pulses

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.36

Hydrodynamic gene delivery: its principles and applications

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.36

Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses

21 January 2018

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Expression of human alpha 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes

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Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

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Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy.

21 January 2018

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Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells

21 January 2018

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Conditional tissue-specific expression of the acid alpha-glucosidase (GAA) gene in the GAA knockout mice: implications for therapy

21 January 2018

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

21 January 2018

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Muscle as a putative producer of acid alpha-glucosidase for glycogenosis type II gene therapy

21 January 2018

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Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer

21 January 2018

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

21 January 2018

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Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation

21 January 2018

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Long-term transgene expression from plasmid DNA gene therapy vectors is negatively affected by CpG dinucleotides

21 January 2018

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

21 January 2018

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Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier

21 January 2018

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Mannose 6-phosphate receptor-mediated transport of sulfamidase across the blood-brain barrier in the newborn mouse

21 January 2018

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

21 January 2018

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Enzyme replacement improves nervous system pathology and function in a mouse model for metachromatic leukodystrophy

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.36

Regional hydrodynamic gene delivery to the rat liver with physiological volumes of DNA solution

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.36

Hydrodynamic gene delivery to the pig liver via an isolated segment of the inferior vena cava

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.36

Direct comparison of hepatocyte-specific expression cassettes following adenoviral and nonviral hydrodynamic gene transfer

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.36

Minicircle: an improved DNA molecule for in vitro and in vivo gene transfer

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.36

Sustained liver-specific transgene expression from the albumin promoter in mice following hydrodynamic plasmid DNA delivery

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.36

Persistent episomal transgene expression in liver following delivery of a scaffold/matrix attachment region containing non-viral vector.

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.36

Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA.

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.36

High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA.

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2009.36

Early onset of lysosomal storage disease in a murine model of mucopolysaccharidosis type VII: undegraded substrate accumulates in many tissues in the fetus and very young MPS VII mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.36

7 January 2021

Lysosomal storage diseases

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.36

7 January 2021

Gene therapy for lysosomal storage diseases

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.36

7 January 2021

Gene therapy progress and prospects: hydrodynamic gene delivery

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.36

7 January 2021

High-level protein secretion into blood circulation after electric pulse-mediated gene transfer into skeletal muscle

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.36

7 January 2021

Long-term secretion of therapeutic proteins from genetically modified skeletal muscles

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.36

7 January 2021

Erythropoietin secretion and physiological effect in mouse after intramuscular plasmid DNA electrotransfer

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.36

7 January 2021

Non-viral transfer approaches for the gene therapy of mucopolysaccharidosis type II (Hunter syndrome).

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.36

7 January 2021

Abnormal immune function in vivo in a murine model of lysosomal storage disease

1 reference

https://api.crossref.org/works/10.1038%2FGT.2009.36

7 January 2021

Identifiers

DOI

10.1038/GT.2009.36

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Dimensions Publication ID

6 December 2017

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