2022
An ELISA-based platform for rapid identification of structure-dependent nucleic acid–protein interactions detects novel DNA triplex interactors
Economos NG, Thapar U, Balasubramanian N, Karras GI, Glazer PM. An ELISA-based platform for rapid identification of structure-dependent nucleic acid–protein interactions detects novel DNA triplex interactors. Journal Of Biological Chemistry 2022, 298: 102398. PMID: 35988651, PMCID: PMC9493393, DOI: 10.1016/j.jbc.2022.102398.Peer-Reviewed Original ResearchMeSH KeywordsDNAEnzyme-Linked Immunosorbent AssayHumansNucleic Acid ConformationPeptide Nucleic AcidsConceptsNucleic acid structuresNucleic acid-protein interactionsNucleotide excision repairSingle-strand annealing repairDouble-strand break intermediatesUnusual nucleic acid structuresNovel interactorNucleic acid interactionsHigh-throughput platformCellular processesFactor localizationAcid structureExcision repairRelevant lociHuman cellsGene editingAcid interactionsInteractorsTherapeutic gene editingNucleic acidsDNA triplexesRapid identificationComparative approachGenomeTriplexesAntispacer peptide nucleic acids for sequence-specific CRISPR-Cas9 modulation
Economos NG, Quijano E, Carufe KEW, Perera JDR, Glazer PM. Antispacer peptide nucleic acids for sequence-specific CRISPR-Cas9 modulation. Nucleic Acids Research 2022, 50: e59-e59. PMID: 35235944, PMCID: PMC9177974, DOI: 10.1093/nar/gkac095.Peer-Reviewed Original Research
2021
Peptide nucleic acids and their role in gene regulation and editing
Perera JDR, Carufe KEW, Glazer PM. Peptide nucleic acids and their role in gene regulation and editing. Biopolymers 2021, 112: e23460. PMID: 34129732, DOI: 10.1002/bip.23460.Peer-Reviewed Original ResearchGene Expression RegulationPeptide Nucleic Acids
2018
In utero nanoparticle delivery for site-specific genome editing
Ricciardi AS, Bahal R, Farrelly JS, Quijano E, Bianchi AH, Luks VL, Putman R, López-Giráldez F, Coşkun S, Song E, Liu Y, Hsieh WC, Ly DH, Stitelman DH, Glazer PM, Saltzman WM. In utero nanoparticle delivery for site-specific genome editing. Nature Communications 2018, 9: 2481. PMID: 29946143, PMCID: PMC6018676, DOI: 10.1038/s41467-018-04894-2.Peer-Reviewed Original ResearchConceptsSite-specific genome editingReversal of splenomegalyPeptide nucleic acidIntra-amniotic administrationBlood hemoglobin levelsMonogenic disordersNanoparticle deliveryPolymeric nanoparticlesPostnatal elevationGestational ageHemoglobin levelsImproved survivalPediatric morbidityDisease improvementHuman β-thalassemiaReticulocyte countNormal organ developmentMouse modelNormal rangeEarly interventionGenome editingOff-target mutationsPostnatal growthGene editingVersatile method
2016
In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery
Bahal R, Ali McNeer N, Quijano E, Liu Y, Sulkowski P, Turchick A, Lu YC, Bhunia DC, Manna A, Greiner DL, Brehm MA, Cheng CJ, López-Giráldez F, Ricciardi A, Beloor J, Krause DS, Kumar P, Gallagher PG, Braddock DT, Mark Saltzman W, Ly DH, Glazer PM. In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery. Nature Communications 2016, 7: 13304. PMID: 27782131, PMCID: PMC5095181, DOI: 10.1038/ncomms13304.Peer-Reviewed Original ResearchConceptsNanoparticle deliveryGene correctionReversal of splenomegalyPeptide nucleic acidLow off-target effectsVivo correctionGenome editingOff-target effectsGene editingHaematopoietic stem cellsNucleic acidsDonor DNAStem cellsΓPNAΒ-thalassaemiaNanoparticlesDeliveryEditingSCF treatmentTriplex formation
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
MicroRNA silencing for cancer therapy targeted to the tumour microenvironment
Cheng CJ, Bahal R, Babar IA, Pincus Z, Barrera F, Liu C, Svoronos A, Braddock DT, Glazer PM, Engelman DM, Saltzman WM, Slack FJ. MicroRNA silencing for cancer therapy targeted to the tumour microenvironment. Nature 2014, 518: 107-110. PMID: 25409146, PMCID: PMC4367962, DOI: 10.1038/nature13905.Peer-Reviewed Original Research
2001
Triplex forming oligonucleotides: sequence-specific tools for gene targeting
Knauert M, Glazer P. Triplex forming oligonucleotides: sequence-specific tools for gene targeting. Human Molecular Genetics 2001, 10: 2243-2251. PMID: 11673407, DOI: 10.1093/hmg/10.20.2243.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsDNADNA RepairForecastingGene TargetingGenetic TherapyHumansMutagenesisNucleic Acid ConformationOligonucleotidesPeptide Nucleic AcidsRecombination, GeneticConceptsHuman gene therapyGene therapy agentsAbility of TFOsTriplex formingGenome modificationGene therapyMammalian cellsGenetic manipulationGene targetingGene expressionPotential applicationsGenetic targetingDuplex DNATherapy agentsMajor grooveLoose canonsHigh specificityGenesRecent studiesTargetingRelated moleculesTFOCellsDevicesDNADirected gene modification via triple helix formation.
Gorman L, Glazer P. Directed gene modification via triple helix formation. 2001, 1: 391-9. PMID: 11899085, DOI: 10.2174/1566524013363771.Peer-Reviewed Original ResearchConceptsGene modificationNon-functional gene productMammalian genesGene productsGenomic DNASingle nucleotideDefective geneTriple helix formationGenetic diseasesTriplex formingGenesHelix formationEfficient targetingNucleic acidsDNAInitial stepGene therapyCorrect sequenceNucleotidesMutationsMoleculesImportant advancesSequenceTargetingModificationGene targeting via triple-helix formation
Casey B, Glazer P. Gene targeting via triple-helix formation. Progress In Nucleic Acid Research And Molecular Biology 2001, 67: 163-192. PMID: 11525382, DOI: 10.1016/s0079-6603(01)67028-4.Peer-Reviewed Original ResearchMeSH KeywordsChromatinDNADNA RepairGene TargetingGenetic TherapyMutagenesisNucleic Acid ConformationPeptide Nucleic AcidsRecombination, GeneticUp-Regulation
1999
Peptide nucleic acid (PNA) binding-mediated induction of human γ-globin gene expression
Wang G, Xu X, Pace B, Dean D, Glazer P, Chan P, Goodman S, Shokolenko I. Peptide nucleic acid (PNA) binding-mediated induction of human γ-globin gene expression. Nucleic Acids Research 1999, 27: 2806-2813. PMID: 10373600, PMCID: PMC148492, DOI: 10.1093/nar/27.13.2806.Peer-Reviewed Original ResearchConceptsGamma-globin gene expressionGamma-globin geneD-loop structureGene expressionHuman γ-globin gene expressionΓ-globin gene expressionGenetic diseasesK562 human erythroleukemia cellsGene expression strategyReporter gene constructsSequence-specific mannerBeta-globin geneHuman erythroleukemia cellsInduction of expressionAdult blood cellsEndogenous genesCommon genetic diseaseGene productsGene constructsExpression strategyErythroleukemia cellsHomopurine/homopyrimidine sequencesHuman diseasesGenesGlobin disorders