2021
The NIH Somatic Cell Genome Editing program
Saha K, Sontheimer EJ, Brooks PJ, Dwinell MR, Gersbach CA, Liu DR, Murray SA, Tsai SQ, Wilson RC, Anderson DG, Asokan A, Banfield JF, Bankiewicz KS, Bao G, Bulte JWM, Bursac N, Campbell JM, Carlson DF, Chaikof EL, Chen ZY, Cheng RH, Clark KJ, Curiel DT, Dahlman JE, Deverman BE, Dickinson ME, Doudna JA, Ekker SC, Emborg ME, Feng G, Freedman BS, Gamm DM, Gao G, Ghiran IC, Glazer PM, Gong S, Heaney JD, Hennebold JD, Hinson JT, Khvorova A, Kiani S, Lagor WR, Lam KS, Leong KW, Levine JE, Lewis JA, Lutz CM, Ly DH, Maragh S, McCray PB, McDevitt TC, Mirochnitchenko O, Morizane R, Murthy N, Prather RS, Ronald JA, Roy S, Roy S, Sabbisetti V, Saltzman WM, Santangelo PJ, Segal DJ, Shimoyama M, Skala MC, Tarantal AF, Tilton JC, Truskey GA, Vandsburger M, Watts JK, Wells KD, Wolfe SA, Xu Q, Xue W, Yi G, Zhou J. The NIH Somatic Cell Genome Editing program. Nature 2021, 592: 195-204. PMID: 33828315, PMCID: PMC8026397, DOI: 10.1038/s41586-021-03191-1.Peer-Reviewed Original ResearchConceptsDownstream functional consequencesGenome modificationHuman genomeGenome editingGenome editorsSomatic cellsHuman cellsFunctional consequencesBiomedical research communityGenomeLarge animalsBiological systemsCellsHuman healthHuman biological systemsEditingVivoAnimal modelsNew therapiesNew opportunitiesWide rangeConsortium
2016
Precise Genome Modification Using Triplex Forming Oligonucleotides and Peptide Nucleic Acids
Bahal R, Gupta A, Glazer P. Precise Genome Modification Using Triplex Forming Oligonucleotides and Peptide Nucleic Acids. Advances In Experimental Medicine And Biology 2016, 93-110. DOI: 10.1007/978-1-4939-3509-3_6.Peer-Reviewed Original ResearchHomologous recombinationSite-specific genome editingPrecise genome modificationGenomic DNA sequencesSpecific genome editingTriplex technologySingle base pair mutationTriplex forming oligonucleotideBase pair mutationGenetic disordersGenome modificationNucleic acidsDefective protein synthesisGenome editingDNA sequencesDifferent genesPair mutationsProtein synthesisMutation sitesGenesSpecific sitesGene replacement therapyExpressionOligonucleotideMutagenesis
2004
Targeted Genome Modification Via Triple Helix Formation
Rogers F, Glazer P. Targeted Genome Modification Via Triple Helix Formation. Cancer Drug Discovery And Development 2004, 27-43. DOI: 10.1007/978-1-59259-777-2_3.Peer-Reviewed Original ResearchDefective geneTargeted genome modificationInhibitors of proteinGene functionGenome modificationGene productsGene expressionTriple helix formationTremendous clinical valueSynthetic oligonucleotidesGenesInherited diseaseHelix formationTreatment of diseasesExpressionDifferent diseasesProteinTherapeutic agentsOligonucleotideTherapeutic useTremendous stridesInhibitorsFunction
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 deliveryOligodeoxyribonucleotidesTriplex 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 moleculesTFOCellsDevicesDNA
2000
Specific Mutations Induced by Triplex-Forming Oligonucleotides in Mice
Vasquez K, Narayanan L, Glazer P. Specific Mutations Induced by Triplex-Forming Oligonucleotides in Mice. Science 2000, 290: 530-533. PMID: 11039937, DOI: 10.1126/science.290.5491.530.Peer-Reviewed Original ResearchConceptsSomatic cellsSpecific genomic sitesEmbryonic stem cell technologyDuplex DNA sequencesGene functionGreater mutation frequenciesGenomic sitesGenome modificationChromosomal copyDNA sequencesSequence-controlled oligomersReporter geneStem cell technologyControl genesGerm-line mutationsGenesSpecific mutationsSupF geneControl oligomersMutationsMutation frequencyTransgenic miceOligonucleotideCellsMutation detection
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 ResearchGenome Modification by Triplex-Forming Oligonucleotides
Vasquez K, Glazer P. Genome Modification by Triplex-Forming Oligonucleotides. Perspectives In Antisense Science 1999, 2: 167-179. DOI: 10.1007/978-1-4615-5177-5_13.Peer-Reviewed Original ResearchSite-specific genome modificationGenome modificationSpecific DNA sequencesExpression of genesLevel of DNAMammalian cellsHomologous recombinationDNA sequencesGene replacementGene expressionLiving cellsTriplex technologyTriplex formingSpecific sitesDNARecombinationExpressionInitial stepCellsMutagenesisGenesMutationsModificationSitesSequence