2022
Gene therapy applications to transfusion medicine
Tabibi S, Gehrie E, Bruscia E, Krause D. Gene therapy applications to transfusion medicine. 2022, 642-647. DOI: 10.1002/9781119719809.ch56.ChaptersGene therapyVector-based gene therapyViral vector-based gene therapyGene therapy applicationsTranscription activator-like effector nucleasesZinc finger nucleasesGene-editing approachesNonviral vectorsGene-editing techniquesGene integrationTherapy applicationsFinger nucleasesEffector nucleasesViral vectorsReplication-competent virusPossible applicationsGenetic materialApplicationsNucleaseTarget cellsVectorCRISPR
2016
Gene therapy applications to transfusion medicine
Gehrie E, Bersenev A, Bruscia E, Krause D, Schulz W. Gene therapy applications to transfusion medicine. 2016, 452-455. DOI: 10.1002/9781119013020.ch38.ChaptersGene therapyTherapeutic genetic materialGene therapy applicationsTranscription activator-like effector nucleasesZinc finger nucleasesPackaging cell lineGene-modified cellsGene-editing approachesNonviral techniquesTherapy applicationsFinger nucleasesEffector nucleasesViral vectorsShort palindromic repeatsVector systemReplication-competent virusPalindromic repeatsHemophilia BPossible applicationsGenetic materialNucleaseApplicationsGenetic diseasesFactor IXVector
2002
Marrow-Derived Cells as Vehicles for Delivery of Gene Therapy to Pulmonary Epithelium
Grove JE, Lutzko C, Priller J, Henegariu O, Theise ND, Kohn DB, Krause DS. Marrow-Derived Cells as Vehicles for Delivery of Gene Therapy to Pulmonary Epithelium. American Journal Of Respiratory Cell And Molecular Biology 2002, 27: 645-651. PMID: 12444022, DOI: 10.1165/rcmb.2002-0056rc.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsBone Marrow TransplantationFemaleGene ExpressionGenetic TherapyGreen Fluorescent ProteinsHematopoietic Stem Cell TransplantationIndicators and ReagentsLuminescent ProteinsLung DiseasesMiceMice, Inbred C57BLProtein PrecursorsProteolipidsRespiratory MucosaRetroviridaeRNA, MessengerConceptsBone marrow-derived stem cellsLung epithelial cellsLung epitheliumEpithelial cellsMarrow-derived stem cellsGene therapyBMSC transplantationLung diseaseFemale miceAirway epitheliumAlveolar spaceLong-term transgene expressionMale marrowPulmonary epitheliumLong-term gene expressionGene therapy applicationsGene expressionPulmonary airwaysAdenoviral vectorEpitheliumMarrowTherapyTransient gene expressionNonviral vectorsStem cells