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
2011
Targeted Gene Modification of Hematopoietic Progenitor Cells in Mice Following Systemic Administration of a PNA-peptide Conjugate
Rogers FA, Lin SS, Hegan DC, Krause DS, Glazer PM. Targeted Gene Modification of Hematopoietic Progenitor Cells in Mice Following Systemic Administration of a PNA-peptide Conjugate. Molecular Therapy 2011, 20: 109-118. PMID: 21829173, PMCID: PMC3255600, DOI: 10.1038/mt.2011.163.Peer-Reviewed Original ResearchConceptsGene modificationGene therapyHematopoietic stem cell gene therapyStem cell gene therapyGenomic modificationsVivo gene therapyCell gene therapyTargeted gene modificationVivo gene modificationHematopoietic progenitor cellsPeptide nucleic acidSystemic administrationBone marrowGene-targeting strategiesProgenitor cellsPrimary recipient miceStem cell mobilizationEx vivo manipulationSickle cell anemiaLymphoid cell lineagesDonor miceRecipient miceHematologic disordersInvasive alternativeCell mobilization
2009
Gene Therapy in Transfusion Medicine
Bruscia E, Krause D. Gene Therapy in Transfusion Medicine. 2009, 936-949. DOI: 10.1002/9781444303513.ch60.Chapters
2005
Engraftment of bone marrow-derived epithelial cells
Van Arnam JS, Herzog E, Grove J, Bruscia E, Ziegler E, Swenson S, Krause DS. Engraftment of bone marrow-derived epithelial cells. Stem Cell Reviews And Reports 2005, 1: 21-27. PMID: 17132871, DOI: 10.1385/scr:1:1:021.Peer-Reviewed Original ResearchConceptsBM-derived cellsEpithelial cellsBM transplantationBone marrow-derived epithelial cellsMarrow-derived epithelial cellsPreclinical mouse modelsType II pneumocytesHematopoietic systemGene therapyFalse-positive cellsGastrointestinal tractHost epithelial cellsMouse modelFemale recipientsPositive cellsBone marrowCre-lox systemTherapeutic potentialTherapeutic useTissue-specific markersTransplantationTherapyPotential gene therapy applicationsCellsHuman diseases
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