2014
Improved bioactivity of G-rich triplex-forming oligonucleotides containing modified guanine bases
Rogers FA, Lloyd JA, Tiwari MK. Improved bioactivity of G-rich triplex-forming oligonucleotides containing modified guanine bases. Artificial DNA PNA & XNA 2014, 5: e27792. PMID: 25483840, PMCID: PMC4014521, DOI: 10.4161/adna.27792.Peer-Reviewed Original ResearchConceptsMobility gel shift assaysDNA double-strand breaksTarget sequenceGel shift assaysDouble-strand breaksGene-targeted mutagenesisActivation of apoptosisG-quadruplex formationGenomic modificationsDNA repairShift assaysReporter geneMolecular mechanismsPolypurine sequenceStrand breaksTriplex technologyTarget siteMutagenesisTriplex structureGenesGuanine baseTriplex formationGuanine basesSequenceAG30
2012
Local Delivery of Gene-Modifying Triplex-Forming Molecules to the Epidermis
Rogers FA, Hu RH, Milstone LM. Local Delivery of Gene-Modifying Triplex-Forming Molecules to the Epidermis. Journal Of Investigative Dermatology 2012, 133: 685-691. PMID: 23014335, PMCID: PMC3532560, DOI: 10.1038/jid.2012.351.Peer-Reviewed Original ResearchConceptsIntradermal deliveryHairless miceTransgenic miceBasal keratinocytesMethod of deliveryNuclei of keratinocytesIntraperitoneal administrationSitu gene correctionBack skinLocal deliveryMiceDisease-causing genesEpidermal keratinocytesKeratinocytesLocal administrationAdministrationDeliverySkinEpidermisDays
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 acidsCellsTargeted Gene Modification of Hematopoietic Progenitor Cells in Mice Following Systemic Administration of a PNA-peptide Conjugate
Rogers FA, Lin SS, Hegan DC, Krause DS, Glazer PM. Targeted Gene Modification of Hematopoietic Progenitor Cells in Mice Following Systemic Administration of a PNA-peptide Conjugate. Molecular Therapy 2011, 20: 109-118. PMID: 21829173, PMCID: PMC3255600, DOI: 10.1038/mt.2011.163.Peer-Reviewed Original ResearchConceptsGene modificationGene therapyHematopoietic stem cell gene therapyStem cell gene therapyGenomic modificationsVivo gene therapyCell gene therapyTargeted gene modificationVivo gene modificationHematopoietic progenitor cellsPeptide nucleic acidSystemic administrationBone marrowGene-targeting strategiesProgenitor cellsPrimary recipient miceStem cell mobilizationEx vivo manipulationSickle cell anemiaLymphoid cell lineagesDonor miceRecipient miceHematologic disordersInvasive alternativeCell mobilization
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
2004
Peptide conjugates for chromosomal gene targeting by triplex-forming oligonucleotides
Rogers FA, Manoharan M, Rabinovitch P, Ward DC, Glazer PM. Peptide conjugates for chromosomal gene targeting by triplex-forming oligonucleotides. Nucleic Acids Research 2004, 32: 6595-6604. PMID: 15602001, PMCID: PMC545466, DOI: 10.1093/nar/gkh998.Peer-Reviewed Original ResearchConceptsMutation frequencyGene-targeted mutagenesisHomeodomain of AntennapediaDNA-binding moleculesChromosomal genesThird helixTarget genesChromosomal DNABiological activityAntpCell-penetrating peptidesGene expressionControl genesTransport peptideCellular uptakeAntigene compoundsGenesConfocal microscopyIntranuclear deliveryTFODose-responsive mannerHomeodomainAntennapediaOligonucleotideMutagenesis