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
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 disordersGenesPromoterGene transfer to ankyrin-deficient bone marrow corrects spherocytosis in vitro
Dooner G, Barker J, Gallagher P, Debatis M, Brown A, Forget B, Becker P. Gene transfer to ankyrin-deficient bone marrow corrects spherocytosis in vitro. Experimental Hematology 2000, 28: 765-774. PMID: 10907638, DOI: 10.1016/s0301-472x(00)00185-5.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsAnkyrinsBlotting, WesternBone MarrowCell LineElectrophoresis, Polyacrylamide GelErythropoietinGene Transfer TechniquesGenetic TherapyHematopoietic Stem CellsHumansIn Vitro TechniquesMiceMice, Inbred BALB CRetroviridaeReverse Transcriptase Polymerase Chain ReactionSpherocytosis, HereditaryConceptsMEL cellsAnkyrin promoterGene transferDependence of expressionMurine bone marrow cellsMurine erythroleukemia cellsNormal murine bone marrow cellsRetroviral vectorsNbs mutantsMutant bone marrowMurine 3T3 fibroblastsNB cellsAnkyrin proteinsMutant cellsPolymerase chain reactionErythroid differentiation culturesHuman hemolytic anemiasColony polymerase chain reactionRT-PCRErythroid expressionBone marrow progenitorsErythroleukemia cellsDifferentiation culturesAnkyrinWestern blot analysis