2009
Targeted correction of a thalassemia-associated β-globin mutation induced by pseudo-complementary peptide nucleic acids
Lonkar P, Kim KH, Kuan JY, Chin JY, Rogers FA, Knauert MP, Kole R, Nielsen PE, Glazer PM. Targeted correction of a thalassemia-associated β-globin mutation induced by pseudo-complementary peptide nucleic acids. Nucleic Acids Research 2009, 37: 3635-3644. PMID: 19364810, PMCID: PMC2699504, DOI: 10.1093/nar/gkp217.Peer-Reviewed Original ResearchConceptsBeta-globin geneNucleotide excision repair factorsAltered helical structureExcision repair factorsSingle base-pair modificationTriplex-forming peptide nucleic acidsPseudo-complementary peptide nucleic acidsDisease-related genesDonor DNA fragmentsComplementary DNA sequenceNucleic acidsProper splicingRepair factorsSite-specific bindingMammalian cellsSite-specific modificationDNA repairDNA sequencesGene targetingSecond intronDNA fragmentsHuman cellsTriple helix formationGene correctionHuman fibroblast cells
2002
Site-directed recombination via bifunctional PNA–DNA conjugates
Rogers FA, Vasquez KM, Egholm M, Glazer PM. Site-directed recombination via bifunctional PNA–DNA conjugates. Proceedings Of The National Academy Of Sciences Of The United States Of America 2002, 99: 16695-16700. PMID: 12461167, PMCID: PMC139206, DOI: 10.1073/pnas.262556899.Peer-Reviewed Original ResearchConceptsSite-directed recombinationDNA repairNucleotide excision repair factorsExcision repair factorsSite-specific DNASite-specific recombinationHuman cell-free extractsDonor DNA fragmentsRepair factorsMammalian cellsGenetic manipulationCell-free extractsTarget genesSite-directed mannerReporter geneStrand invasionPlasmid substratesDNA fragmentsDefective geneDonor DNAWatson-Crick base pairingDuplex DNAGenesTarget plasmidDNA