2024
Peptide Nucleic Acid-Mediated Regulation of CRISPR-Cas9 Specificity
Carufe K, Economos N, Glazer P. Peptide Nucleic Acid-Mediated Regulation of CRISPR-Cas9 Specificity. Nucleic Acid Therapeutics 2024, 34: 245-256. PMID: 39037032, PMCID: PMC11564683, DOI: 10.1089/nat.2024.0007.Peer-Reviewed Original ResearchPeptide nucleic acidProtospacer adjacent motifAllele-specific mannerDegree of homologyWild-type sequencePAM-proximal regionSynthetic peptide nucleic acidOff-target sitesSpacer sequencesAdjacent motifMutant allelesCas9 cuttingBase pairsCas9 activityCRISPR technologyAutosomal dominant diseaseGRNACRISPR applicationsNucleic acidsBinding positionDominant diseaseSequenceDeliberate mismatchGene therapy
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 StatementsConceptsHuman 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 advancesSequenceTargetingModification