2016
In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery
Bahal R, Ali McNeer N, Quijano E, Liu Y, Sulkowski P, Turchick A, Lu YC, Bhunia DC, Manna A, Greiner DL, Brehm MA, Cheng CJ, López-Giráldez F, Ricciardi A, Beloor J, Krause DS, Kumar P, Gallagher PG, Braddock DT, Mark Saltzman W, Ly DH, Glazer PM. In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery. Nature Communications 2016, 7: 13304. PMID: 27782131, PMCID: PMC5095181, DOI: 10.1038/ncomms13304.Peer-Reviewed Original ResearchConceptsNanoparticle deliveryGene correctionReversal of splenomegalyPeptide nucleic acidLow off-target effectsVivo correctionGenome editingOff-target effectsGene editingHaematopoietic stem cellsNucleic acidsDonor DNAStem cellsΓPNAΒ-thalassaemiaNanoparticlesDeliveryEditingSCF treatmentTriplex formation
2010
Functional Analysis of a Novel cis-Acting Regulatory Region within the Human Ankyrin Gene (ANK-1) Promoter
Laflamme K, Owen AN, Devlin EE, Yang MQ, Wong C, Steiner LA, Garrett LJ, Elnitski L, Gallagher PG, Bodine DM. Functional Analysis of a Novel cis-Acting Regulatory Region within the Human Ankyrin Gene (ANK-1) Promoter. Molecular And Cellular Biology 2010, 30: 3493-3502. PMID: 20479128, PMCID: PMC2897556, DOI: 10.1128/mcb.00119-10.Peer-Reviewed Original ResearchMeSH Keywords5' Untranslated RegionsAnimalsAnkyrinsBase SequenceBinding SitesCell-Free SystemConsensus SequenceDNADNA PrimersGene LibraryHumansIn Vitro TechniquesMiceMice, TransgenicMolecular Sequence DataPromoter Regions, GeneticRegulatory Sequences, Nucleic AcidSequence DeletionSequence Homology, Nucleic AcidTATA-Box Binding ProteinTranscription Factor TFIIDTranscription, GeneticConceptsCell-free transcriptionPromoter functionGene promoterDinucleotide deletionANK-1 geneNovel regulatory elementFunctional promoter sequencesNovel functional motifsTransgenic mouse assaysPreinitiation complexRegulatory sequencesPromoter sequencesRegulatory regionsRegulatory elementsFunctional motifsUntranslated regionWild typeFunctional analysisAdditional sequencesDegenerate sequencePromoterMutationsTranscriptionDeletionSequence
2007
Population analysis of the alpha hemoglobin stabilizing protein (AHSP) gene identifies sequence variants that alter expression and function
dos Santos CO, Zhou S, Secolin R, Wang X, Cunha AF, Higgs DR, Kwiatkowski JL, Thein SL, Gallagher PG, Costa FF, Weiss MJ. Population analysis of the alpha hemoglobin stabilizing protein (AHSP) gene identifies sequence variants that alter expression and function. American Journal Of Hematology 2007, 83: 103-108. PMID: 17874450, DOI: 10.1002/ajh.21041.Peer-Reviewed Original Research
2004
Sequences Downstream of the Erythroid Promoter Are Required for High Level Expression of the Human α-Spectrin Gene*
Wong EY, Lin J, Forget BG, Bodine DM, Gallagher PG. Sequences Downstream of the Erythroid Promoter Are Required for High Level Expression of the Human α-Spectrin Gene*. Journal Of Biological Chemistry 2004, 279: 55024-55033. PMID: 15456760, DOI: 10.1074/jbc.m408886200.Peer-Reviewed Original ResearchMeSH KeywordsBase SequenceBinding SitesCell DifferentiationCell MembraneCell NucleusChromatin ImmunoprecipitationCREB-Binding ProteinDeoxyribonuclease IDNADNA PrimersDNA-Binding ProteinsDNA, ComplementaryErythrocytesErythroid-Specific DNA-Binding FactorsEthidiumExonsGATA1 Transcription FactorGenes, ReporterHeLa CellsHumansImmunoprecipitationIntronsK562 CellsLuciferasesModels, GeneticMolecular Sequence DataMutationNuclear ProteinsPlasmidsPromoter Regions, GeneticSpectrinTemperatureTrans-ActivatorsTranscription FactorsTransfectionConceptsErythroid-specific expressionAlpha-spectrin geneGATA-1 sitesCore promoterDNase I hypersensitive sitesElectrophoretic mobility shift assaysChromatin immunoprecipitation assaysMobility shift assaysΑ-spectrin geneThymidine kinase promoterPositive regulatory elementHigh-level expressionGenomic orientationErythroid promoterGATA-1Membrane proteinsHypersensitive sitesImmunoprecipitation assaysRegulatory elementsSequence downstreamShift assaysErythroid differentiationTransfection assaysEnhancer activityReporter gene
2001
Erythrocyte Ankyrin Promoter Mutations Associated with Recessive Hereditary Spherocytosis Cause Significant Abnormalities in Ankyrin Expression*
Gallagher P, Sabatino D, Basseres D, Nilson D, Wong C, Cline A, Garrett L, Bodine D. Erythrocyte Ankyrin Promoter Mutations Associated with Recessive Hereditary Spherocytosis Cause Significant Abnormalities in Ankyrin Expression*. Journal Of Biological Chemistry 2001, 276: 41683-41689. PMID: 11527968, DOI: 10.1074/jbc.m105844200.Peer-Reviewed Original Research
2000
Genomic organization and chromosomal localization of the murine 2 P domain potassium channel gene Kcnk8: conservation of gene structure in 2 P domain potassium channels
Bockenhauer D, Nimmakayalu M, Ward D, Goldstein S, Gallagher P. Genomic organization and chromosomal localization of the murine 2 P domain potassium channel gene Kcnk8: conservation of gene structure in 2 P domain potassium channels. Gene 2000, 261: 365-372. PMID: 11167025, DOI: 10.1016/s0378-1119(00)00492-3.Peer-Reviewed Original ResearchMeSH KeywordsAmino Acid SequenceAnimalsBase SequenceChromosome MappingDNADNA, ComplementaryElectrophysiologyExonsGenesIn Situ Hybridization, FluorescenceIntronsMiceMolecular Sequence DataOocytesPhylogenyPotassium ChannelsPotassium Channels, Tandem Pore DomainProtein Structure, TertiaryRNA, ComplementarySequence Analysis, DNAXenopus laevisConceptsPotassium channel genesDomain potassium channelsChromosomal localizationGene structureOocyte expression systemCDNA sequenceXenopus laevis oocyte expression systemExpression systemChannel genesPotential transmembrane helicesIntron/exon boundariesSingle EF-hand motifOpen reading framePotassium channelsEF-hand motifsEvolutionary conservationGenomic organizationCellular chaperonesGenomic structureComposite cDNAPotential SH3Transmembrane helicesGenome databaseChromosomal genesReading frame
1992
Large numbers of alternatively spliced isoforms of the regulatory region of human erythrocyte ankyrin.
Gallagher PG, Tse WT, Scarpa AL, Lux SE, Forget BG. Large numbers of alternatively spliced isoforms of the regulatory region of human erythrocyte ankyrin. Proceedings Of The Association Of American Physicians 1992, 105: 268-77. PMID: 1309004.Peer-Reviewed Original Research
1991
A splice site mutation of the beta-spectrin gene causing exon skipping in hereditary elliptocytosis associated with a truncated beta-spectrin chain
Gallagher PG, Tse WT, Costa F, Scarpa A, Boivin P, Delaunay J, Forget BG. A splice site mutation of the beta-spectrin gene causing exon skipping in hereditary elliptocytosis associated with a truncated beta-spectrin chain. Journal Of Biological Chemistry 1991, 266: 15154-15159. PMID: 1840591, DOI: 10.1016/s0021-9258(18)98598-4.Peer-Reviewed Original ResearchSpectrin Rouen (beta 220-218), a novel shortened beta-chain variant in a kindred with hereditary elliptocytosis. Characterization of the molecular defect as exon skipping due to a splice site mutation.
Garbarz M, Tse WT, Gallagher PG, Picat C, Lecomte MC, Galibert F, Dhermy D, Forget BG. Spectrin Rouen (beta 220-218), a novel shortened beta-chain variant in a kindred with hereditary elliptocytosis. Characterization of the molecular defect as exon skipping due to a splice site mutation. Journal Of Clinical Investigation 1991, 88: 76-81. PMID: 2056132, PMCID: PMC296005, DOI: 10.1172/jci115307.Peer-Reviewed Original ResearchConceptsBeta-spectrin chainBeta-spectrin geneCDNA amplification productsAmino acidsExon YGenomic DNANucleotide sequencingExample of exonMolecular defectsAnalysis of cDNAAmplification productsHereditary elliptocytosisConsensus splice sitesNovel amino acidCarboxy terminusIntron downstreamSplice siteSplice site mutationSouthern blotExonsExon skipReticulocyte RNACDNAPenultimate exonSite mutationA defect in alpha-spectrin mRNA accumulation in hereditary pyropoikilocytosis.
Gallagher PG, Tse WT, Marchesi SL, Zarkowsky HS, Forget BG. A defect in alpha-spectrin mRNA accumulation in hereditary pyropoikilocytosis. Proceedings Of The Association Of American Physicians 1991, 104: 32-9. PMID: 1845156.Peer-Reviewed Original Research