(Q39872579)

15 September 2017

Cystic fibrosis gene therapy: successes, failures and hopes for the future (English)

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15 September 2017

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Uta Griesenbach

1

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PubMed ID

20477328

15 September 2017

Eric W F W Alton

series ordinal

2

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15 September 2017

1 August 2009

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Expert Review of Respiratory Medicine

15 September 2017

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15 September 2017

3

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15 September 2017

4

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15 September 2017

363-371

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

15 September 2017

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

21 January 2018

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Extracellular barriers in respiratory gene therapy.

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

Effect of genotype on phenotype and mortality in cystic fibrosis: a retrospective cohort study

21 January 2018

1 reference

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

21 January 2018

1 reference

Transient increase in lung epithelial tight junction permeability: an additional mechanism for enhancement of lung transgene expression by perfluorochemical liquids

21 January 2018

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Aerosol delivery of an enhanced helper-dependent adenovirus formulation to rabbit lung using an intratracheal catheter

21 January 2018

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Pulmonary delivery of adenovirus vector formulated with dexamethasone-spermine facilitates homologous vector re-administration.

21 January 2018

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Readministration of adenovirus vector in nonhuman primate lungs by blockade of CD40-CD40 ligand interactions

21 January 2018

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Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors.

21 January 2018

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Protection of Cftr knockout mice from acute lung infection by a helper-dependent adenoviral vector expressing Cftr in airway epithelia

21 January 2018

1 reference

Delivery of CFTR by adenoviral vector to cystic fibrosis mouse lung in a model of chronicPseudomonas aeruginosalung infection

21 January 2018

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Intra-amniotic delivery of CFTR-expressing adenovirus does not reverse cystic fibrosis phenotype in inbred CFTR-knockout mice.

21 January 2018

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Adenovirus-mediated in utero expression of CFTR does not improve survival of CFTR knockout mice

21 January 2018

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Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro.

21 January 2018

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

21 January 2018

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Directed evolution of adeno-associated virus yields enhanced gene delivery vectors

21 January 2018

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Directed evolution of adeno-associated virus to an infectious respiratory virus

21 January 2018

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

21 January 2018

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

21 January 2018

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Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing

21 January 2018

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A shortened adeno-associated virus expression cassette for CFTR gene transfer to cystic fibrosis airway epithelia.

21 January 2018

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Expression of a truncated cystic fibrosis transmembrane conductance regulator with an AAV5-pseudotyped vector in primates

21 January 2018

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Partial correction of the CFTR-dependent ABPA mouse model with recombinant adeno-associated virus gene transfer of truncated CFTR gene

21 January 2018

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Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.

21 January 2018

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High-efficiency promoter-dependent transduction by adeno-associated virus type 6 vectors in mouse lung

21 January 2018

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Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes

21 January 2018

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

21 January 2018

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

21 January 2018

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Analysis of adeno-associated virus progenitor cell transduction in mouse lung.

21 January 2018

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Second-strand genome conversion of adeno-associated virus type 2 (AAV-2) and AAV-5 is not rate limiting following apical infection of polarized human airway epithelia

21 January 2018

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Hybrid adeno-associated virus bearing nonhomologous inverted terminal repeats enhances dual-vector reconstruction of minigenes in vivo.

21 January 2018

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Dual therapeutic utility of proteasome modulating agents for pharmaco-gene therapy of the cystic fibrosis airway.

21 January 2018

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Comparative biology of rAAV transduction in ferret, pig and human airway epithelia.

21 January 2018

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A defective nontransmissible recombinant Sendai virus mediates efficient gene transfer to airway epithelium in vivo

21 January 2018

1 reference

Infection of ciliated cells by human parainfluenza virus type 3 in an in vitro model of human airway epithelium

21 January 2018

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Sendai virus-mediated CFTR gene transfer to the airway epithelium.

21 January 2018

1 reference

Effect of tolerance induction to immunodominant T-cell epitopes of Sendai virus on gene expression following repeat administration to lung

21 January 2018

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

21 January 2018

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Lentiviral vectors pseudotyped with minimal filovirus envelopes increased gene transfer in murine lung

21 January 2018

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Lentivirus vector can be readministered to nasal epithelia without blocking immune responses

21 January 2018

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

21 January 2018

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Ciliated epithelial cell lifespan in the mouse trachea and lung

21 January 2018

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Inducible packaging cells for large-scale production of lentiviral vectors in serum-free suspension culture

21 January 2018

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

21 January 2018

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Use of ultrasound to enhance nonviral lung gene transfer in vivo

21 January 2018

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The role of doxorubicin in non-viral gene transfer in the lung

21 January 2018

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

21 January 2018

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

21 January 2018

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21 January 2018

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

21 January 2018

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

21 January 2018

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Disruption of the CFTR gene produces a model of cystic fibrosis in newborn pigs

21 January 2018

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Repeated Aerosolized AAV-CFTR for Treatment of Cystic Fibrosis: A Randomized Placebo-Controlled Phase 2B Trial

21 January 2018

1 reference

21 January 2018

Identifiers

DOI

10.1586/ERS.09.25

1 reference

15 September 2017

1 reference

PubMed ID

20477328