2011
Targeted Disruption of the CCR5 Gene in Human Hematopoietic Stem Cells Stimulated by Peptide Nucleic Acids
Schleifman EB, Bindra R, Leif J, del Campo J, Rogers FA, Uchil P, Kutsch O, Shultz LD, Kumar P, Greiner DL, Glazer PM. Targeted Disruption of the CCR5 Gene in Human Hematopoietic Stem Cells Stimulated by Peptide Nucleic Acids. Cell Chemical Biology 2011, 18: 1189-1198. PMID: 21944757, PMCID: PMC3183429, DOI: 10.1016/j.chembiol.2011.07.010.Peer-Reviewed Original ResearchConceptsHematopoietic stem cellsHIV-1CCR5 geneHIV-1-infected individualsHIV-1 infectionGene modificationHIV-1 entryCCR5-Delta32 mutationImmune system functionStem cellsCCR5 knockoutMonths posttransplantationChemokine receptorsHuman hematopoietic stem cellsTherapeutic strategiesSubsequent engraftmentGenome modificationProtein levelsHuman cellsTargeted disruptionCCR5Peptide nucleic acidInfectionNucleic acidsCells
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