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
2014
Modified Poly(lactic‐co‐glycolic Acid) Nanoparticles for Enhanced Cellular Uptake and Gene Editing in the Lung
Fields RJ, Quijano E, McNeer NA, Caputo C, Bahal R, Anandalingam K, Egan ME, Glazer PM, Saltzman WM. Modified Poly(lactic‐co‐glycolic Acid) Nanoparticles for Enhanced Cellular Uptake and Gene Editing in the Lung. Advanced Healthcare Materials 2014, 4: 361-366. PMID: 25156908, PMCID: PMC4339402, DOI: 10.1002/adhm.201400355.Peer-Reviewed Original ResearchMeSH KeywordsAmino Acid SequenceAnimalsDNADNA-Binding ProteinsDrug CarriersEpithelial CellsFemaleGene Transfer TechniquesGreen Fluorescent ProteinsLactic AcidLungMacrophages, AlveolarMice, Inbred BALB CMice, TransgenicMolecular Sequence DataNanoparticlesPolyglycolic AcidPolylactic Acid-Polyglycolic Acid CopolymerSurface Properties