2016
Precise Genome Modification Using Triplex Forming Oligonucleotides and Peptide Nucleic Acids
Bahal R, Gupta A, Glazer P. Precise Genome Modification Using Triplex Forming Oligonucleotides and Peptide Nucleic Acids. Advances In Experimental Medicine And Biology 2016, 93-110. DOI: 10.1007/978-1-4939-3509-3_6.Peer-Reviewed Original ResearchHomologous recombinationSite-specific genome editingPrecise genome modificationGenomic DNA sequencesSpecific genome editingTriplex technologySingle base pair mutationTriplex forming oligonucleotideBase pair mutationGenetic disordersGenome modificationNucleic acidsDefective protein synthesisGenome editingDNA sequencesDifferent genesPair mutationsProtein synthesisMutation sitesGenesSpecific sitesGene replacement therapyExpressionOligonucleotideMutagenesis
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
2004
Targeted Genome Modification Via Triple Helix Formation
Rogers F, Glazer P. Targeted Genome Modification Via Triple Helix Formation. Cancer Drug Discovery And Development 2004, 27-43. DOI: 10.1007/978-1-59259-777-2_3.Peer-Reviewed Original ResearchDefective geneTargeted genome modificationInhibitors of proteinGene functionGenome modificationGene productsGene expressionTriple helix formationTremendous clinical valueSynthetic oligonucleotidesGenesInherited diseaseHelix formationTreatment of diseasesExpressionDifferent diseasesProteinTherapeutic agentsOligonucleotideTherapeutic useTremendous stridesInhibitorsFunction
2000
Specific Mutations Induced by Triplex-Forming Oligonucleotides in Mice
Vasquez K, Narayanan L, Glazer P. Specific Mutations Induced by Triplex-Forming Oligonucleotides in Mice. Science 2000, 290: 530-533. PMID: 11039937, DOI: 10.1126/science.290.5491.530.Peer-Reviewed Original ResearchConceptsSomatic cellsSpecific genomic sitesEmbryonic stem cell technologyDuplex DNA sequencesGene functionGreater mutation frequenciesGenomic sitesGenome modificationChromosomal copyDNA sequencesSequence-controlled oligomersReporter geneStem cell technologyControl genesGerm-line mutationsGenesSpecific mutationsSupF geneControl oligomersMutationsMutation frequencyTransgenic miceOligonucleotideCellsMutation detection