Translating Sleeping Beauty transposition into cellular therapies: victories and challenges (Q34127579)

31 July 2017

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title

Translating Sleeping Beauty transposition into cellular therapies: victories and challenges (English)

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31 July 2017

Perry B Hackett

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31 July 2017

Zsuzsanna Izsvák

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31 July 2017

Laurence J N Cooper

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31 July 2017

Zoltán Ivics

series ordinal

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31 July 2017

language of work or name

English

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publication date

1 September 2010

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31 July 2017

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31 July 2017

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31 July 2017

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31 July 2017

page(s)

756-767

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Comparative analysis of transposable element vector systems in human cells

31 July 2017

cites work

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Gene expression in lung and liver after intravenous infusion of polyethylenimine complexes of Sleeping Beauty transposons

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Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system

5 July 2018

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Efficient stable gene transfer into human cells by the Sleeping Beauty transposon vectors.

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Long-term reduction of jaundice in Gunn rats by nonviral liver-targeted delivery of Sleeping Beauty transposon

5 July 2018

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Nanocapsule-delivered Sleeping Beauty mediates therapeutic Factor VIII expression in liver sinusoidal endothelial cells of hemophilia A mice

5 July 2018

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5 July 2018

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5 July 2018

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Applying a "double-feature" promoter to identify cardiomyocytes differentiated from human embryonic stem cells following transposon-based gene delivery.

5 July 2018

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Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

5 July 2018

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Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon system

5 July 2018

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Hybrid lentivirus-transposon vectors with a random integration profile in human cells.

5 July 2018

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Progress toward liver-based gene therapy

5 July 2018

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Image-guided, lobe-specific hydrodynamic gene delivery to swine liver

5 July 2018

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Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

5 July 2018

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Redirecting specificity of T-cell populations for CD19 using the Sleeping Beauty system

5 July 2018

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Sleeping Beauty transposon-mediated engineering of human primary T cells for therapy of CD19+ lymphoid malignancies

5 July 2018

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Preferential delivery of the Sleeping Beauty transposon system to livers of mice by hydrodynamic injection

5 July 2018

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Adoptive transfer of effector CD8+ T cells derived from central memory cells establishes persistent T cell memory in primates

5 July 2018

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Adenovirus vector induced innate immune responses: impact upon efficacy and toxicity in gene therapy and vaccine applications

5 July 2018

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Postintegrative gene silencing within the Sleeping Beauty transposition system

5 July 2018

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5 July 2018

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Erythroid-specific expression of beta-globin by the sleeping beauty transposon for Sickle cell disease

5 July 2018

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Targeted Sleeping Beauty transposition in human cells

5 July 2018

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Transposon-based interferon gamma gene transfer overcomes limitations of episomal plasmid for immunogene therapy of glioblastoma

5 July 2018

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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

5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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Transposons for gene therapy!

5 July 2018

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Molecular analysis of chromosomal rearrangements in mammalian cells after phiC31-mediated integration

5 July 2018

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Sleeping Beauty-based gene therapy with indoleamine 2,3-dioxygenase inhibits lung allograft fibrosis

5 July 2018

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Gene therapy: X-SCID transgene leukaemogenicity

5 July 2018

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Stable gene transfer to human CD34(+) hematopoietic cells using the Sleeping Beauty transposon

5 July 2018

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Modulating target site selection during human immunodeficiency virus DNA integration in vitro with an engineered tethering factor

5 July 2018

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Chimeric Mos1 and piggyBac transposases result in site-directed integration

5 July 2018

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Zinc Finger Tools: custom DNA-binding domains for transcription factors and nucleases

5 July 2018

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Sleeping beauty transposon-mediated nonviral gene therapy

5 July 2018

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Overcoming promoter competition in packaging cells improves production of self-inactivating retroviral vectors

5 July 2018

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

5 July 2018

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

5 July 2018

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Effects of local gene transfer of VEGF on neointima formation after balloon injury in hypercholesterolemic rabbits

5 July 2018

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Neuronal precursor-restricted transduction via in utero CNS gene delivery of a novel bipartite HSV amplicon/transposase hybrid vector

5 July 2018

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RNA as a source of transposase for Sleeping Beauty-mediated gene insertion and expression in somatic cells and tissues

5 July 2018

1 reference

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Silencing and variegation of gammaretrovirus and lentivirus vectors

5 July 2018

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Hyperactive transposase mutants of the Sleeping Beauty transposon

5 July 2018

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Cancer gene discovery in solid tumours using transposon-based somatic mutagenesis in the mouse

5 July 2018

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High-resolution genome-wide mapping of transposon integration in mammals

5 July 2018

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5 July 2018

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Mutational analysis of the N-terminal DNA-binding domain of sleeping beauty transposase: critical residues for DNA binding and hyperactivity in mammalian cells

5 July 2018

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Endothelial targeting of the Sleeping Beauty transposon within lung

5 July 2018

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Development of hyperactive sleeping beauty transposon vectors by mutational analysis

5 July 2018

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Sleeping beauty transposition: biology and applications for molecular therapy

5 July 2018

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Chance or necessity? Insertional mutagenesis in gene therapy and its consequences

5 July 2018

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Targeting survival: integration site selection by retroviruses and LTR-retrotransposons

5 July 2018

1 reference

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

5 July 2018

1 reference

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

5 July 2018

1 reference

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Sustainable correction of junctional epidermolysis bullosa via transposon-mediated nonviral gene transfer

5 July 2018

1 reference

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Transcription start regions in the human genome are favored targets for MLV integration

5 July 2018

1 reference

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Controlling integration specificity of a yeast retrotransposon

5 July 2018

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HIV-1 integration in the human genome favors active genes and local hotspots

5 July 2018

1 reference

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

5 July 2018

1 reference

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Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy

5 July 2018

1 reference

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Sleeping Beauty, a wide host-range transposon vector for genetic transformation in vertebrates

5 July 2018

1 reference

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

5 July 2018

1 reference

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Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

5 July 2018

1 reference

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

5 July 2018

1 reference

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Molecular Reconstruction of Sleeping Beauty, a Tc1-like Transposon from Fish, and Its Transposition in Human Cells

5 July 2018

1 reference

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Expression of Naked Plasmid DNA Injected into the Afferent and Efferent Vessels of Rodent and Dog Livers

5 July 2018

1 reference

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Region-specific recombination and expression are directed by portions of the Drosophila engrailed promoter.

5 July 2018

1 reference

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Gene transfer into coronary arteries of intact animals with a percutaneous balloon catheter

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

Altering the insertional specificity of a Drosophila transposable element

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

Optimization of the PiggyBac transposon system for the sustained genetic modification of human T lymphocytes

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

Stable gene transfer and expression in cord blood-derived CD34+ hematopoietic stem and progenitor cells by a hyperactive Sleeping Beauty transposon system

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

Computer-assisted hydrodynamic gene delivery

28 October 2018

1 reference

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Transcriptional activities of the Sleeping Beauty transposon and shielding its genetic cargo with insulators.

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

Cis-acting gene regulatory activities in the terminal regions of sleeping beauty DNA transposon-based vectors

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

Efficient and stable transgene expression in human embryonic stem cells using transposon-mediated gene transfer

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

CD28 costimulation provided through a CD19-specific chimeric antigen receptor enhances in vivo persistence and antitumor efficacy of adoptively transferred T cells.

28 October 2018

1 reference

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Sleeping Beauty-mediated eNOS gene therapy attenuates monocrotaline-induced pulmonary hypertension in rats

28 October 2018

1 reference

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Pig liver gene therapy by noninvasive interventionist catheterism.

28 October 2018

1 reference

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Hydrodynamic plasmid DNA gene therapy model in liver transplantation

28 October 2018

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Long-term expression and repeated administration of AAV type 1, 2 and 5 vectors in skeletal muscle of immunocompetent adult mice

28 October 2018

1 reference

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Phi c31 integrase induces chromosomal aberrations in primary human fibroblasts

28 October 2018

1 reference

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Combinatorial antiangiogenic gene therapy by nonviral gene transfer using the sleeping beauty transposon causes tumor regression and improves survival in mice bearing intracranial human glioblastoma

28 October 2018

1 reference

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

28 October 2018

1 reference

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phiC31 integrase confers genomic integration and long-term transgene expression in rat retina

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking.

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

Target-site preferences of Sleeping Beauty transposons.

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

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

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

Gene insertion and long-term expression in lung mediated by the Sleeping Beauty transposon system

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

Transposition and targeting of the prokaryotic mobile element IS30 in zebrafish.

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

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

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

Development of catheter-based procedures for transducing the isolated rabbit liver with plasmid DNA.

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

Common physical properties of DNA affecting target site selection of sleeping beauty and other Tc1/mariner transposable elements.

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

The artificial zinc finger coding gene 'Jazz' binds the utrophin promoter and activates transcription

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

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

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3971908

Sleeping beauty vector system moves toward human trials in the United States

28 October 2018

1 reference

12 December 2020

DNA transposons for modification of human primary T lymphocytes

1 reference

12 December 2020

Safety of retroviral gene marking with a truncated NGF receptor

1 reference

12 December 2020

Safety and bioactivity of intracoronary delivery of naked plasmid DNA encoding human atrial natriuretic factor

1 reference

12 December 2020

Identifiers

DOI

10.1002/BIES.201000027

1 reference

31 July 2017

1 reference

31 July 2017

1 reference