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
2001
Intracellular generation of single-stranded DNA for chromosomal triplex formation and induced recombination
Datta H, Glazer P. Intracellular generation of single-stranded DNA for chromosomal triplex formation and induced recombination. Nucleic Acids Research 2001, 29: 5140-5147. PMID: 11812847, PMCID: PMC97609, DOI: 10.1093/nar/29.24.5140.Peer-Reviewed Original ResearchConceptsNovel vector systemMouse cellsInduced recombinationPrimer extension analysisVector systemGenome modificationTriplex formationExtension analysisIntrachromosomal recombinationChromosomal eventsGene expressionSequence insertReporter substrateSuch oligodeoxyribonucleotidesTarget siteSsDNA moleculesIntracellular generationDNARecombinationEfficient intracellular deliveryCellsSuch moleculesSequenceIntracellular deliveryOligodeoxyribonucleotides
1999
Triplex Formation by Oligonucleotides Containing 5-(1-Propynyl)-2‘-deoxyuridine: Decreased Magnesium Dependence and Improved Intracellular Gene Targeting †
Lacroix L, Lacoste J, Reddoch J, Mergny J, Levy D, Seidman M, Matteucci M, Glazer P. Triplex Formation by Oligonucleotides Containing 5-(1-Propynyl)-2‘-deoxyuridine: Decreased Magnesium Dependence and Improved Intracellular Gene Targeting †. Biochemistry 1999, 38: 1893-1901. PMID: 10026270, DOI: 10.1021/bi982290q.Peer-Reviewed Original Research
1997
Potassium-Resistant Triple Helix Formation and Improved Intracellular Gene Targeting by Oligodeoxyribonucleotides Containing 7-Deazaxanthine
Faruqi A, Krawczyk S, Matteucci M, Glazer P. Potassium-Resistant Triple Helix Formation and Improved Intracellular Gene Targeting by Oligodeoxyribonucleotides Containing 7-Deazaxanthine. Nucleic Acids Research 1997, 25: 633-640. PMID: 9016606, PMCID: PMC146453, DOI: 10.1093/nar/25.3.633.Peer-Reviewed Original ResearchConceptsTriple helix-forming oligonucleotidesTriple helix formationGel mobility shift assaysHelix formationMutation reporter geneMobility shift assaysMammalian cellsAnti-gene strategyHigh mutation frequencyShift assaysGene targetingReporter geneGenesPhosphodiester backboneMutation frequencyPsoralen adductsVivo geneTriplex formationPhysiological concentrationsNucleotide chemistry
1996
Mutagenesis in Mammalian Cells Induced by Triple Helix Formation and Transcription-Coupled Repair
Wang G, Seidman M, Glazer P. Mutagenesis in Mammalian Cells Induced by Triple Helix Formation and Transcription-Coupled Repair. Science 1996, 271: 802-805. PMID: 8628995, DOI: 10.1126/science.271.5250.802.Peer-Reviewed Original ResearchConceptsMammalian cellsSufficient binding affinitiesTranscription-coupled repairHuman cell extractsInhibition of transcriptionSimian virus 40 vectorXeroderma pigmentosum groupExcision repairGenetic instabilityTriple helix formationCell extractsTriplex-forming oligonucleotidesGroup B cellsDNA repair synthesisTranscriptionMutagenesisRepair synthesisTriplex DNAHelix formationTriplex formationTriple helixCellsBinding affinitiesTherapeutic applicationsB cells