Current status of gene therapy for inherited lung diseases

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Current status of gene therapy for inherited lung diseases is …
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review articleQ7318358
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P356DOI10.1146/ANNUREV.PHYSIOL.65.092101.142426
P8608Fatcat IDrelease_5boodgtipzhj3i7zmsjwm45lxa
P932PMC publication ID1857313
P698PubMed publication ID12524461
P5875ResearchGate publication ID10954630

P50authorJohn F EngelhardtQ56999987
Ryan DriskellQ43118961
P2093author name stringJohn F Engelhardt
P2860cites workLatent adeno-associated virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate modelQ24527302
Adeno-associated virus type 6 (AAV6) vectors mediate efficient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectorsQ24529153
Adeno-associated virus serotype 4 (AAV4) and AAV5 both require sialic acid binding for hemagglutination and efficient transduction but differ in sialic acid linkage specificityQ24529161
Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virionsQ24531466
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapyQ24535690
Site-specific integration by adeno-associated virusQ24558738
Parvovirus replicationQ24634739
Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeuticsQ28141424
Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesisQ28213353
Inhibition of recombinant adeno-associated virus (rAAV) transduction by bronchial secretions from cystic fibrosis patientsQ45739682
Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerizationQ45742577
Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapyQ28297851
Isolation of a common receptor for Coxsackie B viruses and adenoviruses 2 and 5Q28303765
In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vectorQ29547503
A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiencyQ33340517
Cystic fibrosis and the salt controversyQ33544405
Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2.Q33781976
In vitro and in vivo biology of recombinant adenovirus vectors with E1, E1/E2A, or E1/E4 deleted.Q33782356
Successful readministration of adeno-associated virus vectors to the mouse lung requires transient immunosuppression during the initial exposureQ33786029
Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypesQ33793801
Incorporation of adeno-associated virus in a calcium phosphate coprecipitate improves gene transfer to airway epithelia in vitro and in vivo.Q33795565
Impaired intracellular trafficking of adeno-associated virus type 2 vectors limits efficient transduction of murine fibroblastsQ33796712
Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transferQ33802811
CD40 ligand-dependent activation of cytotoxic T lymphocytes by adeno-associated virus vectors in vivo: role of immature dendritic cellsQ33810726
Generation of an adenovirus vector lacking E1, e2a, E3, and all of E4 except open reading frame 3.Q33815920
Repeated delivery of adeno-associated virus vectors to the rabbit airwayQ33823405
Concatamerization of adeno-associated virus circular genomes occurs through intermolecular recombination.Q33823450
Jaagsiekte sheep retrovirus Env protein stabilizes retrovirus vectors against inactivation by lung surfactant, centrifugation, and freeze-thaw cycling.Q33854928
Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirusQ33901983
An animal model for cystic fibrosis made by gene targetingQ33970549
Expression of the cystic fibrosis gene in adult human lungQ34126273
Defective fluid transport by cystic fibrosis airway epitheliaQ34134003
Gene therapy: trials and tribulationsQ34186665
Gene transfer into the airway epithelium of animals by targeting the polymeric immunoglobulin receptorQ34199152
Ribozyme gene therapy: applications for molecular medicineQ34234190
Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus.Q34433170
Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2.Q34486856
AlphaVbeta5 integrin: a co-receptor for adeno-associated virus type 2 infectionQ34486863
Neuroepithelial bodies of pulmonary airways serve as a reservoir of progenitor cells capable of epithelial regenerationQ34506533
Gene repair and transposon-mediated gene therapyQ34565709
Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins.Q34777289
Ablation of E2A in recombinant adenoviruses improves transgene persistence and decreases inflammatory response in mouse liverQ35559973
Actin filaments mediate DNA fiber formation in chronic inflammatory airway diseaseQ35774309
Trans-splicing vectors expand the utility of adeno-associated virus for gene therapyQ35799349
Extensive posttranscriptional deletion of the coding sequences for part of nucleotide-binding fold 1 in respiratory epithelial mRNA transcripts of the cystic fibrosis transmembrane conductance regulator gene is not associated with the clinical manifQ35821409
Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenovirusesQ35837026
A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activationQ45742580
Structural analysis of adeno-associated virus transduction circular intermediates.Q45747262
Quantitative analysis of the packaging capacity of recombinant adeno-associated virusQ45766162
High doses of a helper-dependent adenoviral vector yield supraphysiological levels of alpha1-antitrypsin with negligible toxicityQ45855434
Viral receptors and vector purification: new approaches for generating clinical-grade reagentsQ45857978
Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial.Q45861736
The extra- and intracellular barriers to lipid and adenovirus-mediated pulmonary gene transfer in native sheep airway epitheliumQ45862101
Increasing epithelial junction permeability enhances gene transfer to airway epithelia In vivo.Q45865025
A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosisQ45866575
Methods for construction of adenovirus vectorsQ45866700
Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectorsQ45867424
Recombinant IL-12 prevents formation of blocking IgA antibodies to recombinant adenovirus and allows repeated gene therapy to mouse lung.Q45867442
Progenitor cells of the adult human airway involved in submucosal gland development.Q45867961
Prolonged transgene expression in cotton rat lung with recombinant adenoviruses defective in E2a.Q45870000
Inhibitory effect of cystic fibrosis sputum on adenovirus-mediated gene transfer in cultured epithelial cellsQ45871368
Aerosol and intravenous transfection of human alpha 1-antitrypsin gene to lungs of rabbitsQ45873756
Stem cell niches in the mouse airway.Q45878035
Aerosol and lobar administration of a recombinant adenovirus to individuals with cystic fibrosis. II. Transfection efficiency in airway epitheliumQ45878973
Hepatic gene therapy: persistent expression of human alpha 1-antitrypsin in mice after direct gene delivery in vivoQ45879739
Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparisonQ45880308
Loss of binding and entry of liposome-DNA complexes decreases transfection efficiency in differentiated airway epithelial cellsQ45880343
A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis.Q45881607
Evidence for safety and efficacy of DOTAP cationic liposome mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosisQ45881610
Safety of a single aerosol administration of escalating doses of the cationic lipid GL-67/DOPE/DMPE-PEG5000 formulation to the lungs of normal volunteersQ45884078
Lentiviral vectors for gene therapy of cystic fibrosis.Q45885318
Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity.Q45885499
Lef1 transcription factor expression defines airway progenitor cell targets for in utero gene therapy of submucosal gland in cystic fibrosisQ45887687
Cellular heterogeneity of CFTR expression and function in the lung: implications for gene therapy of cystic fibrosis.Q45889649
Basolateral localization of fiber receptors limits adenovirus infection from the apical surface of airway epitheliaQ50109548
Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosisQ57416213
Na+ and Cl? conductances in airway epithelial cells: increased Na+ conductance in cystic fibrosisQ59278426
Stepwise dismantling of adenovirus 2 during entry into cellsQ64377608
Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant miceQ64377665
Helper-dependent adenoviral vectorsQ64379053
Partial correction of endogenous DeltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicingQ64379396
A phase I study of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator gene to a lung segment of individuals with cystic fibrosisQ64381103
Vector-specific complementation profiles of two independent primary defects in cystic fibrosis airwaysQ64382112
Aerosol administration of a recombinant adenovirus expressing CFTR to cystic fibrosis patients: a phase I clinical trialQ64382613
Detailed analysis of structures and formulations of cationic lipids for efficient gene transfer to the lungQ64382719
Modification of nasal epithelial potential differences of individuals with cystic fibrosis consequent to local administration of a normal CFTR cDNA adenovirus gene transfer vectorQ64383094
Submucosal glands are the predominant site of CFTR expression in the human bronchusQ67469589
Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosisQ71660966
Improvements in cationic liposomes for in vivo gene transferQ71732071
The inhibitory role of CpG immunostimulatory motifs in cationic lipid vector-mediated transgene expression in vivoQ73022551
Human alpha-defensin 1 (HNP-1) inhibits adenoviral infection in vitroQ74338160
Efficient and persistent gene transfer of AAV-CFTR in maxillary sinusQ77292224
Expression of alpha v beta 5 integrin is necessary for efficient adenovirus-mediated gene transfer in the human airway.Q35847135
Transient subversion of CD40 ligand function diminishes immune responses to adenovirus vectors in mouse liver and lung tissuesQ35868592
Transduction by adeno-associated virus vectors in the rabbit airway: efficiency, persistence, and readministration.Q35889903
A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signalQ35915922
Efficient expression of CFTR function with adeno-associated virus vectors that carry shortened CFTR genesQ36287507
Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectorsQ36734619
Feline immunodeficiency virus vectors persistently transduce nondividing airway epithelia and correct the cystic fibrosis defectQ36807130
Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expressionQ36830429
Repeat administration of an adenovirus vector encoding cystic fibrosis transmembrane conductance regulator to the nasal epithelium of patients with cystic fibrosisQ37353301
Lack of high affinity fiber receptor activity explains the resistance of ciliated airway epithelia to adenovirus infectionQ37371791
Incorporation of adenovirus in calcium phosphate precipitates enhances gene transfer to airway epithelia in vitro and in vivoQ37387177
Expression of the cystic fibrosis transmembrane conductance regulator gene in the respiratory tract of normal individuals and individuals with cystic fibrosisQ37557674
The serous cellQ37876724
Limited entry of adenovirus vectors into well-differentiated airway epithelium is responsible for inefficient gene transferQ39582545
Influence of cell polarity on retrovirus-mediated gene transfer to differentiated human airway epithelia.Q39583034
Humoral immunity to adeno-associated virus type 2 vectors following administration to murine and nonhuman primate muscleQ39589663
Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors.Q39682659
A clinical inflammatory syndrome attributable to aerosolized lipid-DNA administration in cystic fibrosisQ40603327
Development of a FLP/frt system for generating helper-dependent adenoviral vectorsQ40805792
Alpha 1-antitrypsin deficiency: an overviewQ40857527
Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicingQ40906944
Binding and uptake of cationic lipid:pDNA complexes by polarized airway epithelial cellsQ40972916
Retrovirus-mediated gene transfer in lungs of living fetal sheep.Q40980092
The effect of mucolytic agents on gene transfer across a CF sputum barrier in vitroQ41052060
Advances in adenoviral vectors: from genetic engineering to their biologyQ41101850
Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site-specific fashion in an immortalized epithelial cell line.Q41171031
Recombinant adenovirus deleted of all viral genes for gene therapy of cystic fibrosisQ41226051
Targeting cell-specific gene expression with an adenovirus vector containing the lacZ gene under the control of the CFTR promoterQ41244961
Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoterQ41573620
Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosisQ41607205
Adenoviral vectors for gene transferQ41628950
In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epitheliumQ41644008
Efficient CFTR expression from AAV vectors packaged with promoters--the second generationQ41688873
Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus.Q41746617
Adenovirus-mediated transfer of a recombinant alpha 1-antitrypsin gene to the lung epithelium in vivoQ41894646
Polarity influences the efficiency of recombinant adenoassociated virus infection in differentiated airway epithelia.Q42466029
Inefficient gene transfer by adenovirus vector to cystic fibrosis airway epithelia of mice and humansQ42476540
Direct gene transfer of human CFTR into human bronchial epithelia of xenografts with E1-deleted adenoviruses.Q42483331
Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis.Q42483788
Evidence for stem-cell niches in the tracheal epitheliumQ42506114
Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung.Q42524846
Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis.Q43535009
EGTA enhancement of adenovirus-mediated gene transfer to mouse tracheal epithelium in vivoQ43553736
A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung diseaseQ43670160
Complement activation by recombinant adenoviruses.Q43861771
Altered fluid transport across airway epithelium in cystic fibrosisQ43917311
CFTR gene transduction in neonatal rabbits using an adeno-associated virus (AAV) vectorQ45279254
Perfluorochemical liquid enhances adeno-associated virus-mediated transgene expression in lungsQ45739293
P921main subjectgene therapyQ213901
lung diseaseQ3392853
P304page(s)585-612
P577publication date2002-05-01
P1433published inAnnual Review of PhysiologyQ567364
P1476titleCurrent status of gene therapy for inherited lung diseases
P478volume65

Reverse relations

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