2023
Phenotypic and proteomic characterization of the human erythroid progenitor continuum reveal dynamic changes in cell cycle and in metabolic pathways
Papoin J, Yan H, Leduc M, Le Gall M, Narla A, Palis J, Steiner L, Gallagher P, Hillyer C, Gautier E, Mohandas N, Blanc L. Phenotypic and proteomic characterization of the human erythroid progenitor continuum reveal dynamic changes in cell cycle and in metabolic pathways. American Journal Of Hematology 2023, 99: 99-112. PMID: 37929634, PMCID: PMC10877306, DOI: 10.1002/ajh.27145.Peer-Reviewed Original ResearchConceptsErythroid progenitor differentiationCell cycleErythroid progenitorsProgenitor differentiationMass spectrometry-based proteomicsFurther functional analysisSpectrometry-based proteomicsHuman erythroid progenitorsProtein machineryErythroid progenitor proliferationTerminal erythropoiesisProteomic characterizationHematopoietic stem cellsProteomic dataProgenitor populationsHuman erythropoiesisReticulocyte maturationFunctional analysisErythroid lineageOxidative phosphorylationProgenitor proliferationErythroid disordersMetabolic pathwaysAbsolute expressionStem cells
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
2007
Characterization of Regulatory Elements in the Slc4a1 (Band 3) Locus To Create Safe and Effective Vectors for Globin Gene Therapy
Harrow F, Battle S, Seidel N, Cline A, Gallagher P, Bodine D. Characterization of Regulatory Elements in the Slc4a1 (Band 3) Locus To Create Safe and Effective Vectors for Globin Gene Therapy. Blood 2007, 110: 3740. DOI: 10.1182/blood.v110.11.3740.3740.Peer-Reviewed Original ResearchGene therapyLentiviral vectorsEffective gene therapyGene therapy vectorsHigh-throughput real-time PCRGlobin gene therapyTherapy vectorsGlobin vectorsEcotropic envelopeNew vectorEffective vectorLentivirus vectorHematopoietic stem cellsEnhancer-blocking activityHigh titersSafe deliverySpleen focusTherapeutic levelsErythroid-specific promoterStem cellsΒ-globin promoterLower virus titersNovel strategySafe expressionReporter gene expressionIdentification of a TFIID Binding Sequence That Increases Expression from the Ankyrin-1 Promoter: Application to Globin Gene Therapy
Laflamme K, Elnitski L, Wong C, Mary Y, Gallagher P, Bodine D. Identification of a TFIID Binding Sequence That Increases Expression from the Ankyrin-1 Promoter: Application to Globin Gene Therapy. Blood 2007, 110: 3741. DOI: 10.1182/blood.v110.11.3741.3741.Peer-Reviewed Original ResearchGene therapyEffective gene therapyGlobin gene therapyDifferent tissue-specific promotersTissue-specific promotersVector copiesViral vectorsDouble-copy vectorVirus vectorsHematopoietic stem cellsSpecific promotersPromoter libraryLibrary of promotersRecombinant virusesStem cellsInefficient productionRecent demonstrationReporter geneTG dinucleotideInsertional activationHigh titersΓ-globin expressionTherapeutic levelsVectorLocus control region
2001
Development of a Stable Retrovirus Vector Capable of Long‐Term Expression of γ‐Globin mRNA in Mouse Erythrocytes
SABATINO D, SEIDEL N, CLINE A, ANDERSON S, GALLAGHER P, BODINE D. Development of a Stable Retrovirus Vector Capable of Long‐Term Expression of γ‐Globin mRNA in Mouse Erythrocytes. Annals Of The New York Academy Of Sciences 2001, 938: 246-261. PMID: 11458514, DOI: 10.1111/j.1749-6632.2001.tb03595.x.Peer-Reviewed Original ResearchConceptsGamma-globin geneLocus control regionGamma-globin mRNARetrovirus vectorHematopoietic stem cellsGene promoterHuman gamma-globin geneMouse alpha-globin mRNAGlobin gene promoterAlpha-globin mRNAStem cellsMature red blood cellsNumber-dependent expressionMouse progenitor cellsΓ-globin mRNAGlobin genesControl regionLevel of expressionStable gene transferGenesGene transferPromoterProgenitor cellsGene therapyMRNA
2000
Long-term expression of γ-globin mRNA in mouse erythrocytes from retrovirus vectors containing the human γ-globin gene fused to the ankyrin-1 promoter
Sabatino D, Seidel N, Aviles-Mendoza G, Cline A, Anderson S, Gallagher P, Bodine D. Long-term expression of γ-globin mRNA in mouse erythrocytes from retrovirus vectors containing the human γ-globin gene fused to the ankyrin-1 promoter. Proceedings Of The National Academy Of Sciences Of The United States Of America 2000, 97: 13294-13299. PMID: 11069298, PMCID: PMC27218, DOI: 10.1073/pnas.230453097.Peer-Reviewed Original ResearchConceptsGlobin genesRetrovirus vectorHematopoietic stem cellsGene promoterHuman γ-globin genesMouse alpha-globin mRNACis-acting regulatory sequencesGlobin gene promoterMouse hematopoietic stem cellsΓ-globin geneGamma-globin geneAlpha-globin mRNAStem cellsMature red blood cellsNumber-dependent expressionIntact proviral sequencesΓ-globin mRNARegulatory sequencesLevel of expressionGlobin mRNARed cell disordersGenesPromoter