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 expression
2004
Sequences Downstream of the Erythroid Promoter Promote High-Level Expression of the Human α-Spectrin Gene by Providing Boundary Activity and Positive Regulatory Elements.
Gallagher P, Nilson D, Lin J, Bodine D. Sequences Downstream of the Erythroid Promoter Promote High-Level Expression of the Human α-Spectrin Gene by Providing Boundary Activity and Positive Regulatory Elements. Blood 2004, 104: 1572. DOI: 10.1182/blood.v104.11.1572.1572.Peer-Reviewed Original ResearchPositive regulatory elementGATA-1 sitesAγ-globin geneRegulatory elementsIntron 1Exon 1Gene expressionLuc activitySplice siteGATA-1 activityErythroid-specific expressionHypersensitive site mappingΒ-globin promoterΑ-spectrin geneK562 cellsTransient transfection assaysMutant exon 1Γ-globin proteinMouse linesHigh-level expressionChromatin modifications