2009
A Modified ANK-1 Promoter Directs Uniform, Copy Number Dependent Gamma Globin Gene Expression at Therapeutic Levels of a in a Lentivirus Vector.
Harrow F, Cline A, Seidel N, Persons D, Gallagher P, Bodine D. A Modified ANK-1 Promoter Directs Uniform, Copy Number Dependent Gamma Globin Gene Expression at Therapeutic Levels of a in a Lentivirus Vector. Blood 2009, 114: 3565. DOI: 10.1182/blood.v114.22.3565.3565.Peer-Reviewed Original ResearchBeta-like globin genesMouse alpha-globinGamma-globin geneGamma-globin mRNAGlobin gene expressionLocus control regionGamma-globin expressionGlobin genesAlpha-globinHematopoietic stem cellsGene expressionEnhancer elementsMouse alpha-globin mRNAHuman gamma-globin geneBeta-globin locus control regionGamma-globin gene expressionSequence variantsTranscription initiation complexLCR elementsBeta-like globinCopy number-dependent expressionBinding of TFIIDAlpha-globin mRNACis-acting enhancer elementPromoter sequence variants
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 disordersGenesPromoterA Minimal Ankyrin Promoter Linked to a Human γ-Globin Gene Demonstrates Erythroid Specific Copy Number Dependent Expression with Minimal Position or Enhancer Dependence in Transgenic Mice*
Sabatino D, Wong C, Cline A, Pyle L, Garrett L, Gallagher P, Bodine D. A Minimal Ankyrin Promoter Linked to a Human γ-Globin Gene Demonstrates Erythroid Specific Copy Number Dependent Expression with Minimal Position or Enhancer Dependence in Transgenic Mice*. Journal Of Biological Chemistry 2000, 275: 28549-28554. PMID: 10878017, DOI: 10.1074/jbc.m004043200.Peer-Reviewed Original ResearchConceptsAnkyrin promoterPosition-independent expressionErythroid cellsGlobin genesGlobin mRNAHuman γ-globin genesMouse alpha-globin mRNABeta-globin locus control regionAdult erythroid cellsANK-1 geneΓ-globin geneCopy number-dependent expressionLocus control regionErythroid-specific expressionAlpha-globin mRNAFetal erythroid cellsNumber-dependent expressionGlobin mRNA expressionErythrocyte membrane skeletonTransgene copy numberHS2 enhancerTransgenic linesGlobin transgenesControl regionTransgenic mice