Non-viral vectors in cystic fibrosis gene therapy: recent developments and future prospects (Q37526991)

20 August 2017

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title

Non-viral vectors in cystic fibrosis gene therapy: recent developments and future prospects (English)

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20 August 2017

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I A Pringle

1

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20 August 2017

S C Hyde

series ordinal

2

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20 August 2017

D R Gill

series ordinal

3

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20 August 2017

language of work or name

English

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publication date

1 August 2009

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Expert Opinion on Biological Therapy

20 August 2017

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20 August 2017

9

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20 August 2017

8

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20 August 2017

991-1003

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Incidence, population, and survival of cystic fibrosis in the UK, 1968-95. UK Cystic Fibrosis Survey Management Committee

20 August 2017

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Cystic fibrosis clinical trials

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Identification of the cystic fibrosis gene: genetic analysis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Identification of the cystic fibrosis gene: chromosome walking and jumping

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

ABC transporters: the power to change

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Mucociliary clearance in cystic fibrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

In vivo airway surface liquid Cl- analysis with solid-state electrodes

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

CFTR as a cAMP-dependent regulator of sodium channels

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Molecular insights into the physiology of the 'thin film' of airway surface liquid

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Gene therapy for human genetic disease?

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Expression of the cystic fibrosis gene in adult human lung

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Normalization of raised sodium absorption and raised calcium-mediated chloride secretion by adenovirus-mediated expression of cystic fibrosis transmembrane conductance regulator in primary human cystic fibrosis airway epithelial cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Adeno-associated virus-targeted disruption of the CFTR gene in cloned ferrets.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Production of CFTR-null and CFTR-DeltaF508 heterozygous pigs by adeno-associated virus-mediated gene targeting and somatic cell nuclear transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Transfection efficiency and toxicity following delivery of naked plasmid DNA and cationic lipid–DNA complexes to ovine lung segments

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Optimizing aerosol gene delivery and expression in the ovine lung

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Electroporation enhances reporter gene expression following delivery of naked plasmid DNA to the lung.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Detection of plasmid DNA vectors following gene transfer to the murine airways.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Electrohydrodynamic comminution: a novel technique for the aerosolisation of plasmid DNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Gene transfer to the lung: lessons learned from more than 2 decades of CF gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Evidence for safety and efficacy of DOTAP cationic liposome mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Compacted DNA nanoparticles administered to the nasal mucosa of cystic fibrosis subjects are safe and demonstrate partial to complete cystic fibrosis transmembrane regulator reconstitution

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Adenovirus infection in cystic fibrosis patients: implications for the use of adenoviral vectors for gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

A concentrated and stable aerosol formulation of cationic lipid:DNA complexes giving high-level gene expression in mouse lung

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Comparison of DNA-lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelia in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

A clinical inflammatory syndrome attributable to aerosolized lipid-DNA administration in cystic fibrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Transfection of airway epithelium by stable PEGylated poly-L-lysine DNA nanoparticles in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Minimal toxicity of stabilized compacted DNA nanoparticles in the murine lung

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Role of nucleolin in human parainfluenza virus type 3 infection of human lung epithelial cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Polyethylenimine shows properties of interest for cystic fibrosis gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

A comparison of linear and branched polyethylenimine (PEI) with DCChol/DOPE liposomes for gene delivery to epithelial cells in vitro and in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Methodological optimization of polyethylenimine (PEI)-based gene delivery to the lungs of mice via aerosol application.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Chloride accumulation and swelling in endosomes enhances DNA transfer by polyamine-DNA polyplexes

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Exploring polyethylenimine-mediated DNA transfection and the proton sponge hypothesis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Lactosylated polyethylenimine for gene transfer into airway epithelial cells: role of the sugar moiety in cell delivery and intracellular trafficking of the complexes

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

In vivo gene delivery in the mouse lung with lactosylated polyethylenimine, questioning the relevance of in vitro experiments

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

An integrin-targeted non-viral vector for pulmonary gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

A receptor-targeted nanocomplex vector system optimized for respiratory gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Formation of LID vector complexes in water alters physicochemical properties and enhances pulmonary gene expression in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Analysis and optimization of the cationic lipid component of a lipid/peptide vector formulation for enhanced transfection in vitro and in vivo.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Evaluation of a porcine model for pulmonary gene transfer using a novel synthetic vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Using magnetic forces to enhance non-viral gene transfer to airway epithelium in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Use of ultrasound to enhance nonviral lung gene transfer in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Electroporation for targeted gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Electroporation as a method for high-level nonviral gene transfer to the lung

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

In vivo electroporation and ubiquitin promoter--a protocol for sustained gene expression in the lung.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Electroporation-mediated transfer of plasmids to the lung results in reduced TLR9 signaling and inflammation

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Progress and prospects: the design and production of plasmid vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

The phiC31 integrase system for gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Targeted Sleeping Beauty transposition in human cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Lung-directed gene therapy in mice using the nonviral Sleeping Beauty transposon system

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor 1alpha promoter.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Minicircle-DNA production by site specific recombination and protein-DNA interaction chromatography.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

A human epithelium-specific vector optimized in rat pneumocytes for lung gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Targeting expression of a transgene to the airway surface epithelium using a ciliated cell-specific promoter

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Expression of CFTR from a ciliated cell-specific promoter is ineffective at correcting nasal potential difference in CF mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Development of lentiviral vectors with regulated respiratory epithelial expression in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Gene transfer of CFTR to airway epithelia: low levels of expression are sufficient to correct Cl- transport and overexpression can generate basolateral CFTR.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

High and sustained transgene expression in vivo from plasmid vectors containing a hybrid ubiquitin promoter

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

A Toll-like receptor recognizes bacterial DNA

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Contribution of Toll-like receptor 9 signaling to the acute inflammatory response to nonviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Immune effects and therapeutic applications of CpG motifs in bacterial DNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Systematic identification of sense-antisense transcripts in mammalian cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903055029

10.1517/14712590903055029

21 January 2018

Identifiers

DOI

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20 August 2017

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