References
- WelshMJSmithAEMolecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosisCell1993737125112547686820
- Cystic Fibrosis FoundationPatient Registry: Annual Data Report 2010Bethesda, MDCystic Fibrosis Foundation2010
- TarranRButtonBPicherMNormal and cystic fibrosis airway surface liquid homeostasis. The effects of phasic shear stress and viral infectionsJ Biol Chem200528042357513575916087672
- MuhlebachMSNoahTLEndotoxin activity and inflammatory markers in the airways of young patients with cystic fibrosisAm J Respir Crit Care Med2002165791191511934713
- KarpCLFlickLMParkKWDefective lipoxin-mediated anti-inflammatory activity in the cystic fibrosis airwayNat Immunol20045438839215034576
- HartlDGrieseMKapplerMPulmonary T(H)2 response in Pseudomonas aeruginosa-infected patients with cystic fibrosisJ Allergy Clin Immunol2006117120421116387607
- AshlockMAOlsonERTherapeutics development for cystic fibrosis: a successful model for a multisystem genetic diseaseAnnu Rev Med20116210712521226613
- KitsonCAngelBJuddDThe extra-and intracellular barriers to lipid and adenovirus-mediated pulmonary gene transfer in native sheep airway epitheliumGene Ther19996453454610476213
- ZabnerJRamsayBWMeekerDPRepeat administration of an adenovirus vector encoding cystic fibrosis transmembrane conductance regulator to the nasal epithelium of patients with cystic fibrosisJ Clin Invest1996976150415118617884
- SchwarztDAQuinnTJThornePSSayeedSYiAKKriegAMCpG motifs in bacterial DNA cause inflammation in the lower respiratory tractJ Clin Invest1997100168739202058
- CrystalRGMcElvaneyNGRosenfeldMAAdministration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosisNat Gene1994814251
- WaltersRWGrunstTBergelsonJMBasolateral localization of fiber receptors limits adenovirus infection from the apical surface of airway epitheliaJ Biol Chem199927415102191022610187807
- ParsonsDWGrubbBRJohnsonLGBoucherRCEnhanced in vivo airway gene transfer via transient modification of host barrier properties with a surface-active agentHum Gene Ther1998918266122729874264
- WeissDJBeckettTBonneauLYoungJKollsJKWangGTransient increase in lung epithelial tight junction permeability: an additional mechanism for enhancement of lung transgene expression by perfluorochemical liquidsMol Ther20038692793514664795
- ZhangLWangDFischerHEfficient expression of CFTR function with adeno-associated virus vectors that carry shortened CFTR genesProc Natl Acad Sci U S A1998951710158101639707617
- SongYLouHHBoyerJLFunctional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicingHum Gene Ther200920326728119257851
- FerrariSGriesenbachUIidaASendai virus-mediated CFTR gene transfer to the airway epitheliumGene Ther2007141371137917597790
- BishopAEPulmonary epithelial stem cellsCell Prolif2004371899614871239
- Hacein-Bey-AbinaSGarrigueAWangGPInsertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1J Clin Invest200811893132314218688285
- JohnsonLGOlsenJCNaldiniLBoucherRCPseudotyped human lentiviral vector-mediated gene transfer to airway epithelia in vivoGene Ther20007756857410819571
- GriesenbachUFerrariSGeddesDMAltonEWFWGene therapy progress and prospects: cystic fibrosisGene Ther20029201344135012364999
- DelendaCLentiviral vectors: optimization of packaging, transduction and gene expressionJ Gene Med20046 Suppl 1S125S13814978756
- MitomoKGriesenbachUInoueMToward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopesMol Ther20101861173118220332767
- HydeSCPringleIAAbdullahSCpG-free plasmids confer reduced inflammation and sustained pulmonary gene expressionNat Biotechnol200826554955118438402
- EngelhardtJFYankaskasJRErnstSASubmucosal glands are the predominant site of CFTR expression in the human bronchusNat Genet1992232402481285365
- EngelhardtJFZepedaMCohnJAYankaskasJRWilsonJMExpression of the cystic fibrosis gene in adult human lungJ Clin Invest19949327377497509347
- SinnPLAnthonyRMMcCrayPBJrGenetic therapies for cystic fibrosis lung diseaseHum Mol Genet201120R1R79R8621422098
- DaviesJCAltonEWGene therapy for cystic fibrosisProc Am Thorac Soc20107640841421030522
- GriesenbachUSmithSNFarleyRSinghCAltonEWValidation of nasal potential difference measurements in gut-corrected CF knockout miceAm J Respir Cell Mol Biol200839449049618458238
- KnowlesMRParadisoAMBoucherRCIn vivo nasal potential difference: techniques and protocols for assessing efficacy of gene transfer in cystic fibrosisHum Gene Ther1995644454557542031
- AccursoFJRoweSMClancyJPEffect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutationN Engl J Med2010363211991200321083385
- JohnsonLGBoylesSEWilsonJBoucherRCNormalization 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 cellsJ Clin Invest1995953137713827533790
- BronsveldIMekusFBijmanJChloride conductance and genetic background modulate the cystic fibrosis phenotype of Delta F508 homozygous twins and siblingsJ Clin Invest2001108111705171511733566
- SimmondsNJD’SouzaLRoughtonMAltonEWDaviesJCHodsonMECystic fibrosis and survival to 40 years: a study of cystic fibrosis transmembrane conductance regulator functionEur Respir J20113751076108220847077
- ZabnerJCoutureLAGregoryRJGrahamSMSmithAEWelshMJAdenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosisCell19937522072167691415
- KnowlesMRHohnekerKWZhouZA controlled study of adenoviral-vector mediated gene transfer in the nasal epithelium of patients with cystic fibrosisN Engl J Med1995333138238317544439
- HayJGMcElvaneyNGHerenaJCrystalRGModification of nasal epithelial potential differences of individuals with cystic fibrosis consequent to local administration of a normal CFTR cDNA adenovirus gene transfer vectorHum Gene Ther1995611148714968573621
- BellonGMichel-CalemardLThouvenotDAerosol administration of a recombinant adenovirus expressing CFTR to cystic fibrosis patients: a phase I clinical trialHum Gene Ther19978115258989991
- HarveyBGLeopoldPLHackettNRAirway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirusJ Clin Invest199910491245125510545523
- ZuckermanJBRobinsonCBMcCoyKSA phase I study of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator gene to a lung segment of individuals with cystic fibrosisHum Gene Ther199910182973298510609658
- JosephPMO’SullivanBPLapeyAAerosol and lobar administration of a recombinant adenovirus to individuals with cystic fibrosis. I. Methods, safety, and clinical implicationsHum Gene Ther200112111369138211485629
- PerriconeMAMorrisJEPavelkaKAerosol and lobar administration of a recombinant adenovirus to individuals with cystic fibrosis. II. Transfection efficiency in airway epitheliumHum Gene Ther200112111383139411485630
- WagnerJAMessnerAHMoranMLSafety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinusLaryngoscope19991092 Pt 126627410890777
- AitkenMLMossRBWaltzDAA phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung diseaseHum Gene Ther200112151907191611589832
- WagnerJANepomucenoIBMessnerAHA phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomiesHum Gene Ther200213111349135912162817
- FlotteTRZeitlinPLReynoldsTCPhase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical studyHum Gene Ther200314111079108812885347
- MossRBRodmanDSpencerLTRepeated 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 trialChest2004125250952114769732
- MossRBMillaCColomboJRepeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trialHum Gene Ther200718872673217685853
- CaplenNJAltonEWMiddletonPGLiposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosisNat Med19951139467584951
- GillDRSouthernKWMoffordKAA placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosisGene Ther1997431992099135733
- PorteousDJDorinJRMcLachlanGEvidence for safety and efficacy of DOTAP cationic liposome mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosisGene Ther1997432102189135734
- HydeSCSouthernKWGileadiURepeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosisGene Ther20007131156116510918483
- ZabnerJChengSHMeekerDComparison of DNA–lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelia in vivoJ Clin Invest19971006152915379294121
- AltonEWSternMFarleyRCationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trialLancet1999353915794795410459902
- RuizFEClancyJPPerriconeMAA clinical inflammatory syndrome attributable to aerosolized lipid–DNA administration in cystic fibrosisHum Gene Ther200112775176111339892
- KonstanMWDavisPBWagenerJSCompacted DNA nanoparticles administered to the nasal mucosa of cystic fibrosis subjects are safe and demonstrate partial to complete cystic fibrosis transmembrane regulator reconstitutionHum Gene Ther200415121255126915684701