(Q36226779)

12 August 2017

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title

A novel gene expression system: non-viral gene transfer for hemophilia as model systems (English)

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12 August 2017

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Carol H Miao

1

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12 August 2017

publication date

1 January 2005

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12 August 2017

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12 August 2017

volume

54

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12 August 2017

page(s)

143-177

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Genetic dissection of a mammalian replicator in the human beta-globin locus

12 August 2017

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Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A.

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Introns increase transcriptional efficiency in transgenic mice

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The insulation of genes from external enhancers and silencing chromatin

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Heteroduplex screening for molecular defects in factor IX genes from haemophilia B families

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Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo

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Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo

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Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver

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Circular YAC vectors containing a small mammalian origin sequence can associate with the nuclear matrix

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Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression

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In vivo footprinting analysis of the hepatic control region of the human apolipoprotein E/C-I/C-IV/C-II gene locus

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Structure of the hepatic control region of the human apolipoprotein E/C-I gene locus

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Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element.

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

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The beta-globin locus control region versus gene therapy vectors: a struggle for expression.

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Gene therapy for the treatment of hemophilia B using PINC-formulated plasmid delivered to muscle with electroporation

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7 January 2021

Upstream and intronic regulatory sequences interact in the activation of the glutamine synthetase promoter

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Matrix attachment region binding protein MFP1 is localized in discrete domains at the nuclear envelope

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Regulatory functions of 3'UTRs.

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

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Immune evasion by muscle-specific gene expression in dystrophic muscle

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An improved plasmid DNA expression vector for direct injection into skeletal muscle

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7 January 2021

Analysis of muscle creatine kinase regulatory elements in recombinant adenoviral vectors

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7 January 2021

Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector

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7 January 2021

Gene therapy: a 2001 perspective

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7 January 2021

Herpes simplex virus latent RNA (LAT) is not required for latent infection in the mouse

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7 January 2021

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

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7 January 2021

Methylation of episomal plasmids as a barrier to transient gene expression via a synthetic delivery vector

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7 January 2021

A herpes simplex virus transcript abundant in latently infected neurons is dispensable for establishment of the latent state

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7 January 2021

Micrococcal nuclease digestion of nuclei reveals extended nucleosome ladders having anomalous DNA lengths for chromatin assembled on non-replicating plasmids in transfected cells

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7 January 2021

Persistence of plasmid DNA and expression in rat brain cells in vivo

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7 January 2021

Increased in vitro and in vivo transgene expression levels mediated through cis-acting elements

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7 January 2021

DNA methylation directs a time-dependent repression of transcription initiation.

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7 January 2021

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

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7 January 2021

Long-term hepatic adenovirus-mediated gene expression in mice following CTLA4Ig administration

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7 January 2021

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

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7 January 2021

Locus control region of the human CD2 gene in a lentivirus vector confers position-independent transgene expression

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7 January 2021

Toll-like receptor expression reveals CpG DNA as a unique microbial stimulus for plasmacytoid dendritic cells which synergizes with CD40 ligand to induce high amounts of IL-12

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7 January 2021

Isolation of human sequences that replicate autonomously in human cells

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7 January 2021

Genetic mechanisms of age regulation of human blood coagulation factor IX

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7 January 2021

Role of intron I in expression of the human factor IX gene

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7 January 2021

Noninvasive gene delivery to the liver by mechanical massage

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7 January 2021

Mechanism of liver gene transfer by mechanical massage

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7 January 2021

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

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7 January 2021

Rat growth hormone gene introns stimulate nucleosome alignment in vitro and in transgenic mice

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7 January 2021

Enhanced expression of transgenes from adeno-associated virus vectors with the woodchuck hepatitis virus posttranscriptional regulatory element: implications for gene therapy

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7 January 2021

Differential nuclear localization and nuclear matrix association of the splicing factors PSF and PTB

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7 January 2021

Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro

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7 January 2021

Long-term and therapeutic-level hepatic gene expression of human factor IX after naked plasmid transfer in vivo

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7 January 2021

High-Level Factor VIII Gene ExpressionIn VivoAchieved by Nonviral Liver-Specific Gene Therapy Vectors

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7 January 2021

Gene therapy by intramuscular injection of plasmid DNA: studies on firefly luciferase gene expression in mice

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7 January 2021

High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors

1 reference

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7 January 2021

Suppression of the tumorigenic growth of Burkitt's lymphoma cells in immunodeficient mice by cytokine gene transfer using EBV-derived episomal expression vectors

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7 January 2021

Prospects for gene therapy of haemophilia

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

Circular YAC vectors containing short mammalian origin sequences are maintained under selection as HeLa episomes

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https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

In vitro polyadenylation is stimulated by the presence of an upstream intron

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7 January 2021

Transitions in distinct histone H3 methylation patterns at the heterochromatin domain boundaries

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

The canine factor VIII 3'-untranslated region and a concatemeric hepatocyte nuclear factor 1 regulatory element enhance factor VIII transgene expression in vivo

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

Liver-directed gene therapy: a retroviral vector with a complete LTR and the ApoE enhancer-alpha 1-antitrypsin promoter dramatically increases expression of human alpha 1-antitrypsin in vivo

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

Heterologous introns can enhance expression of transgenes in mice

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

Influence of promoter and WHV post-transcriptional regulatory element on AAV-mediated transgene expression in the rat brain

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

A vector based on the SV40 origin of replication and chromosomal S/MARs replicates episomally in CHO cells

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7 January 2021

Lentiviral vectors for enhanced gene expression in human hematopoietic cells

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7 January 2021

CpG methylation of a plasmid vector results in extended transgene product expression by circumventing induction of immune responses

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7 January 2021

Context Dependence of Different Modules for Posttranscriptional Enhancement of Gene Expression from Retroviral Vectors

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7 January 2021

Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity.

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7 January 2021

The cytomegalovirus enhancer: a pan-active control element in transgenic mice

1 reference

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7 January 2021

Translation of vascular endothelial growth factor mRNA by internal ribosome entry: implications for translation under hypoxia

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7 January 2021

Epstein-Barr virus/human vector provides high-level, long-term expression of alpha1-antitrypsin in mice

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7 January 2021

A novel DNA replication origin identified in the human heat shock protein 70 gene promoter

1 reference

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7 January 2021

Targeted transfection by femtosecond laser

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

Persistent hepatic expression of human apo A-I after transfer with a helper-virus independent adenoviral vector

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

A mutation in the 3' untranslated region of the factor IX gene in four families with hemophilia B.

1 reference

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7 January 2021

Construction of human factor IX expression vectors in retroviral vector frames optimized for muscle cells

1 reference

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7 January 2021

Three-dimensional visualization of transcription sites and their association with splicing factor-rich nuclear speckles

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https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

In vivo activity for initiation of DNA replication resides in a transcribed region of the human genome

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

Woodchuck hepatitis virus post-transcriptional regulation element enhances transgene expression from adenovirus vectors

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

Naked DNA transfer of Factor VIII induced transgene-specific, species-independent immune response in hemophilia A mice

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

Complete and sustained phenotypic correction of hemophilia B in mice following hepatic gene transfer of a high-expressing human factor IX plasmid

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

Contribution of Plasmid DNA to Inflammation in the Lung after Administration of Cationic Lipid:pDNA Complexes

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7 January 2021

CpG-depleted plasmid DNA vectors with enhanced safety and long-term gene expression in vivo

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7 January 2021

Reduced inflammatory response to plasmid DNA vectors by elimination and inhibition of immunostimulatory CpG motifs

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https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

Long-term expression of human alpha1-antitrypsin gene in mouse liver achieved by intravenous administration of plasmid DNA using a hydrodynamics-based procedure

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors.

1 reference

https://api.crossref.org/works/10.1016%2FS0065-2660%2805%2954007-0

7 January 2021

10.1016/S0065-2660(05)54007-0

Identifiers

DOI

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12 August 2017

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