2015
Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium
McNeer NA, Anandalingam K, Fields RJ, Caputo C, Kopic S, Gupta A, Quijano E, Polikoff L, Kong Y, Bahal R, Geibel JP, Glazer PM, Saltzman WM, Egan ME. Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium. Nature Communications 2015, 6: 6952. PMID: 25914116, PMCID: PMC4480796, DOI: 10.1038/ncomms7952.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsCell LineChloridesCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDNA-Binding ProteinsGenetic TherapyHigh-Throughput Nucleotide SequencingHumansLactic AcidMice, Inbred C57BLNanoparticlesPeptide Nucleic AcidsPolyglycolic AcidPolylactic Acid-Polyglycolic Acid CopolymerPolymersRespiratory MucosaConceptsFacile genome engineeringVivo gene deliveryBiodegradable polymer nanoparticlesTransient gene expressionNanoparticle systemsGene deliveryPolymer nanoparticlesGene correctionGenome engineeringNanoparticlesOff-target effectsPeptide nucleic acidLethal genetic disorderNucleic acidsDonor DNATarget effectsIntranasal deliveryDeliveryCystic fibrosisEngineeringOligonucleotideChloride effluxHuman cellsAirway epitheliumLung tissue
2012
Surface modified poly(β amino ester)-containing nanoparticles for plasmid DNA delivery
Fields RJ, Cheng CJ, Quijano E, Weller C, Kristofik N, Duong N, Hoimes C, Egan ME, Saltzman WM. Surface modified poly(β amino ester)-containing nanoparticles for plasmid DNA delivery. Journal Of Controlled Release 2012, 164: 41-48. PMID: 23041278, PMCID: PMC3625648, DOI: 10.1016/j.jconrel.2012.09.020.Peer-Reviewed Original ResearchConceptsTransfection efficiencyPDNA loadingNovel nanoparticle systemPlasmid DNA loadingGene delivery vehiclesGene therapy applicationsPlasmid DNA deliverySurface coatingSurface chargePBAE polymerCoated nanoparticlesNanoparticle systemsLiposomal vectorsUnmodified particlesDNA deliveryDNA loadingPDNA deliveryTherapy applicationsCellular internalizationDelivery vehiclesUnmodified NPsPBAEIntracellular uptakeBlended particlesNanoparticles