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 expressionIdentification of a TFIID Binding Sequence That Increases Expression from the Ankyrin-1 Promoter: Application to Globin Gene Therapy
Laflamme K, Elnitski L, Wong C, Mary Y, Gallagher P, Bodine D. Identification of a TFIID Binding Sequence That Increases Expression from the Ankyrin-1 Promoter: Application to Globin Gene Therapy. Blood 2007, 110: 3741. DOI: 10.1182/blood.v110.11.3741.3741.Peer-Reviewed Original ResearchGene therapyEffective gene therapyGlobin gene therapyDifferent tissue-specific promotersTissue-specific promotersVector copiesViral vectorsDouble-copy vectorVirus vectorsHematopoietic stem cellsSpecific promotersPromoter libraryLibrary of promotersRecombinant virusesStem cellsInefficient productionRecent demonstrationReporter geneTG dinucleotideInsertional activationHigh titersΓ-globin expressionTherapeutic levelsVectorLocus control region