(Q35904424)

11 August 2017

1 reference

title

Gene therapy for cystic fibrosis: an example for lung gene therapy (English)

1 reference

11 August 2017

0 references

0 references

Griesenbach U

1

1 reference

11 August 2017

Geddes DM

series ordinal

2

1 reference

11 August 2017

Alton EW

series ordinal

3

1 reference

11 August 2017

publication date

1 October 2004

1 reference

11 August 2017

1 reference

11 August 2017

volume

11 Suppl 1

1 reference

11 August 2017

page(s)

S43-50

1 reference

https://scigraph.springernature.com/pub.10.1038/sj.gt.3302368

11 August 2017

exact match

0 references

cites work

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Expression of the cystic fibrosis gene in adult human lung

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Immunological hurdles to lung gene therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Delivery of gene transfer vectors to lung: obstacles and the role of adjunct techniques for airway administration

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Barriers to and new approaches for gene therapy and gene delivery in cystic fibrosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Retargeting the coxsackievirus and adenovirus receptor to the apical surface of polarized epithelial cells reveals the glycocalyx as a barrier to adenovirus-mediated gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Role of alveolar macrophages in rapid elimination of adenovirus vectors administered to the epithelial surface of the respiratory tract

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Aerosol Delivery of aβ-Galactosidase Adenoviral Vector to the Lungs of Rodents

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Safety of local delivery of low- and intermediate-dose adenovirus gene transfer vectors to individuals with a spectrum of morbid conditions

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Aerosol and lobar administration of a recombinant adenovirus to individuals with cystic fibrosis. I. Methods, safety, and clinical implications

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Basolateral localization of fiber receptors limits adenovirus infection from the apical surface of airway epithelia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Enhancement of adenovirus-mediated gene transfer to the airways by DEAE dextran and sodium caprate in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirus

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Reduced inflammation and improved airway expression using helper-dependent adenoviral vectors with a K18 promoter.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

RGD inclusion in VP3 provides adeno-associated virus type 2 (AAV2)-based vectors with a heparan sulfate-independent cell entry mechanism

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Successful transgene expression with serial doses of aerosolized rAAV2 vectors in rhesus macaques

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Global non-viral gene transfer to the primate brain following intravenous administration.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

A New Sendai Virus Vector Deficient in the Matrix Gene Does Not Form Virus Particles and Shows Extensive Cell-to-Cell Spreading

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Apical barriers to airway epithelial cell gene transfer with amphotropic retroviral vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Recovery of airway cystic fibrosis transmembrane conductance regulator function in mice with cystic fibrosis after single-dose lentivirus-mediated gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Lentivirus vectors pseudotyped with filoviral envelope glycoproteins transduce airway epithelia from the apical surface independently of folate receptor alpha

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Pseudotyped lentivirus vectors derived from simian immunodeficiency virus SIVagm with envelope glycoproteins from paramyxovirus

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Sugar-mediated uptake of glycosylated polylysines and gene transfer into normal and cystic fibrosis airway epithelial cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Uptake of plasmid/glycosylated polymer complexes and gene transfer efficiency in differentiated airway epithelial cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Lactosylated poly-L-lysine targets a potential lactose receptor in cystic fibrosis and non-cystic fibrosis airway epithelial cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Functional evidence of CFTR gene transfer in nasal epithelium of cystic fibrosis mice in vivo following luminal application of DNA complexes targeted to the serpin-enzyme complex receptor

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

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/10.1038%2FSJ.GT.3302368

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/10.1038%2FSJ.GT.3302368

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/10.1038%2FSJ.GT.3302368

Targeting transgene expression for cystic fibrosis gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Gene transfer into the airway epithelium of animals by targeting the polymeric immunoglobulin receptor

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Targeted replacement of normal and mutant CFTR sequences in human airway epithelial cells using DNA fragments

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Control of cystic fibrosis transmembrane conductance regulator expression by BAP31.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

An algorithm for finding protein-DNA binding sites with applications to chromatin-immunoprecipitation microarray experiments

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

Gene therapy for cystic fibrosis with aerosolized adenovirus-CFTR: characterization of the aerosol and scintigraphic determination of lung deposition in baboons

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302368

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/10.1038%2FSJ.GT.3302368

Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice

21 January 2018

1 reference

12 December 2020

Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus

1 reference

12 December 2020

The cells of the pulmonary airways

1 reference

12 December 2020

Pre-clinical and clinical endpoint assays for cystic fibrosis gene therapy

1 reference

12 December 2020

Identifying treatments that halt progression of pulmonary disease in cystic fibrosis

1 reference

12 December 2020

Transduction by adeno-associated virus vectors in the rabbit airway: efficiency, persistence, and readministration.

1 reference

12 December 2020

Partial correction of endogenous DeltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing

1 reference

12 December 2020

Deposition and expression of aerosolized rAAV vectors in the lungs of Rhesus macaques

1 reference

12 December 2020

Repeated delivery of adeno-associated virus vectors to the rabbit airway

1 reference

12 December 2020

Adenovirus-mediated persistent cystic fibrosis transmembrane conductance regulator expression in mouse airway epithelium

1 reference

12 December 2020

Identifiers

DOI

10.1038/SJ.GT.3302368

1 reference

11 August 2017

1 reference