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
2006
Lentivirus Vectors Containing a Band 3/γ-Globin Gene Flanked by Distinct Insulator Elements Are Resistant to Gene Silencing in Primary Mouse Erythroid Cells.
Harrow F, Frazar T, Seidel N, Gallagher P, Bodine D. Lentivirus Vectors Containing a Band 3/γ-Globin Gene Flanked by Distinct Insulator Elements Are Resistant to Gene Silencing in Primary Mouse Erythroid Cells. Blood 2006, 108: 3260. DOI: 10.1182/blood.v108.11.3260.3260.Peer-Reviewed Original ResearchΓ-globin geneLocus control regionInsulator elementsΓ-globinΓ-globin mRNAB3 promoterStart siteΓ-globin gene expressionMouse hematopoietic progenitor cellsLike globin genesAbundant membrane proteinErythroid-specific expressionTranscription start siteΓ-globin expressionMouse erythroid cellsCorrect start sitesHigh-throughput real-time PCRHypersensitive site 4Transgenic miceΒ-globin geneMultiple upstream sitesInsulator activityGlobin genesHematopoietic stem cellsMembrane proteins