(Q24684749)

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

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

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Sleeping Beauty transposon system

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based on heuristic

inferred from title

Perry B Hackett

11

object named as

Perry B Hackett

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9

object named as

R Scott McIvor

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Lalitha R Belur

3

object named as

Lalitha R Belur

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author name string

Elena L Aronovich

1

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Jason B Bell

2

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Roland Gunther

4

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Brenda Koniar

5

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David C C Erickson

6

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Patricia A Schachern

7

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Ilze Matise

8

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Chester B Whitley

10

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language of work or name

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publication date

May 2007

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Journal of Gene Medicine

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9

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5

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403-15

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describes a project that uses

1 reference

Europe PubMed Central

11 June 2022

https://www.ebi.ac.uk/europepmc/webservices/rest/PMC1868578/fullTextXML

based on heuristic

inferred from PubMed Central ID database lookup

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

7 April 2017

Murine mucopolysaccharidosis type I: targeted disruption of the murine alpha-L-iduronidase gene

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

7 April 2017

Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

7 April 2017

Limitations of enzyme replacement therapy: current and future

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

Cell and gene-based therapies for the lysosomal storage diseases

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

Gene therapy for lysosomal storage diseases.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

Sustained FVIII expression and phenotypic correction of hemophilia A in neonatal mice using an endothelial-targeted sleeping beauty transposon

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

Sleeping beauty transposon-mediated gene therapy for prolonged expression

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

Awakening gene therapy with Sleeping Beauty transposons

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

Cardiac manifestations in the mouse model of mucopolysaccharidosis I.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

Kupffer cells and not liver sinusoidal endothelial cells prevent lentiviral transduction of hepatocytes

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

Integration and long-term expression in xenografted human glioblastoma cells using a plasmid-based transposon system

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

Gene transfer into genomes of human cells by the sleeping beauty transposon system.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

Progress and prospects: naked DNA gene transfer and therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

Structure-function analysis of the inverted terminal repeats of the sleeping beauty transposon

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

Retroviral vector design studies toward hematopoietic stem cell gene therapy for mucopolysaccharidosis type I.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

Trafficking of lysosomal enzymes.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 September 2017

Structure-based prediction of insertion-site preferences of transposons into chromosomes

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

30 May 2018

Excision of Sleeping Beauty transposons: parameters and applications to gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

30 May 2018

Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

30 May 2018

Cyclophosphamide diminishes inflammation and prolongs transgene expression following delivery of adenoviral vectors to mouse liver and lung.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

30 May 2018

Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

Nonviral in vivo gene transfer in the mucopolysaccharidosis I murine model

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

Dynamic gene expression after systemic delivery of plasmid DNA as determined by in vivo bioluminescence imaging.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

Mammalian mutagenesis using a highly mobile somatic Sleeping Beauty transposon system.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

Phenotypic correction and long-term expression of factor VIII in hemophilic mice by immunotolerization and nonviral gene transfer using the Sleeping Beauty transposon system

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

Partial correction of the alpha-galactosidase A deficiency and reduction of glycolipid storage in Fabry mice using synthetic vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

Helper-Independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

In vivo correction of murine tyrosinemia type I by DNA-mediated transposition.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

Transcriptional activation of a hybrid promoter composed of cytomegalovirus enhancer and beta-actin/beta-globin gene in glomerular epithelial cells in vivo.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

Humoral immune response limits gene therapy in canine MPS I.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

Long-term outcome of Hurler syndrome following bone marrow transplantation.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1868578

28 November 2018

Enzyme replacement therapy: from concept to clinical practice

1 reference

https://pubmed.ncbi.nlm.nih.gov/17407189

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Inborn errors of mucopolysaccharide metabolism

1 reference

https://pubmed.ncbi.nlm.nih.gov/17407189

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Genetically corrected autologous stem cells engraft, but host immune responses limit their utility in canine alpha-L-iduronidase deficiency

1 reference

https://pubmed.ncbi.nlm.nih.gov/17407189

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Beta-glucuronidase deficiency mucopolysaccharidosis: methods for enzymatic diagnosis.

1 reference

https://pubmed.ncbi.nlm.nih.gov/17407189

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Enzyme replacement therapy in mucopolysaccharidosis type II (Hunter syndrome): a preliminary report

1 reference

https://api.crossref.org/works/10.1002%2FJGM.1028

7 January 2021

based on heuristic

inferred from DOI database lookup

Identifiers

10.1002/JGM.1028

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