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