2021
Nanoparticles for delivery of agents to fetal lungs
Ullrich SJ, Freedman-Weiss M, Ahle S, Mandl HK, Piotrowski-Daspit AS, Roberts K, Yung N, Maassel N, Bauer-Pisani T, Ricciardi AS, Egan ME, Glazer PM, Saltzman WM, Stitelman DH. Nanoparticles for delivery of agents to fetal lungs. Acta Biomaterialia 2021, 123: 346-353. PMID: 33484911, PMCID: PMC7962939, DOI: 10.1016/j.actbio.2021.01.024.Peer-Reviewed Original ResearchConceptsFetal lungCellular uptakeIntra-amniotic routeRoute of deliveryCongenital lung diseaseDelivery of agentsIntra-amniotic deliveryRelative cellular uptakeNanoparticlesFetal treatmentDiaphragmatic herniaLung diseaseFetal therapyLung tissueFetal miceIntravenous deliveryCystic fibrosisLungLung therapyInterventional technologiesTherapeutic agentsEndothelial cellsCell populationsEffective targetingTherapy
2018
In utero nanoparticle delivery for site-specific genome editing
Ricciardi AS, Bahal R, Farrelly JS, Quijano E, Bianchi AH, Luks VL, Putman R, López-Giráldez F, Coşkun S, Song E, Liu Y, Hsieh WC, Ly DH, Stitelman DH, Glazer PM, Saltzman WM. In utero nanoparticle delivery for site-specific genome editing. Nature Communications 2018, 9: 2481. PMID: 29946143, PMCID: PMC6018676, DOI: 10.1038/s41467-018-04894-2.Peer-Reviewed Original ResearchConceptsSite-specific genome editingReversal of splenomegalyPeptide nucleic acidIntra-amniotic administrationBlood hemoglobin levelsMonogenic disordersNanoparticle deliveryPolymeric nanoparticlesPostnatal elevationGestational ageHemoglobin levelsImproved survivalPediatric morbidityDisease improvementHuman β-thalassemiaReticulocyte countNormal organ developmentMouse modelNormal rangeEarly interventionGenome editingOff-target mutationsPostnatal growthGene editingVersatile method
2015
Life-Long Transgene Expression in Skeletal Muscle Without Transduction of Satellite Cells Following Embryonic Myogenic Progenitor Transduction by Lentivirus Administered in Utero
Stitelman DH, Brazelton TR, Endo M, Bora A, Traas J, Zoltick PW, Flake AW. Life-Long Transgene Expression in Skeletal Muscle Without Transduction of Satellite Cells Following Embryonic Myogenic Progenitor Transduction by Lentivirus Administered in Utero. Stem Cells And Development 2015, 24: 1878-1887. PMID: 25915576, PMCID: PMC4533031, DOI: 10.1089/scd.2014.0585.Peer-Reviewed Original ResearchConceptsSatellite cellsMarker gene expressionMyogenic stem cellsMammalian myogenesisTransgene expressionMuscle progenitorsPrimitive streakMyogenic progenitorsCommon progenitorGene expressionMarker transgeneAdult muscleGene transferTransductionStem cellsCell transductionDermomyotomeProgenitorsSkeletal muscleMuscle fibersSustained transgene expressionExpressionDermatomyotomeCellsRecent evidence
2010
Robust In Vivo Transduction of Nervous System and Neural Stem Cells by Early Gestational Intra Amniotic Gene Transfer Using Lentiviral Vector
Stitelman DH, Endo M, Bora A, Muvarak N, Zoltick PW, Flake AW, Brazelton TR. Robust In Vivo Transduction of Nervous System and Neural Stem Cells by Early Gestational Intra Amniotic Gene Transfer Using Lentiviral Vector. Molecular Therapy 2010, 18: 1615-1623. PMID: 20571539, PMCID: PMC2956924, DOI: 10.1038/mt.2010.125.Peer-Reviewed Original ResearchConceptsNeural stem cellsAdult neural stem cellsCell typesGene transferStem cellsGreen fluorescent protein marker geneProtein marker geneNervous system developmentNeural cell typesMajor neural cell typesLentiviral vectorsNeural cell populationsMajor cell typesAdult nervous systemGene functionMarker genesEmbryonic day 8Subventricular zoneEntire nervous systemNervous systemVivo transductionCell populationsFuture clinical applicationsGenetic disordersNeural groove
2009
The developmental stage determines the distribution and duration of gene expression after early intra-amniotic gene transfer using lentiviral vectors
Endo M, Henriques-Coelho T, Zoltick PW, Stitelman DH, Peranteau WH, Radu A, Flake AW. The developmental stage determines the distribution and duration of gene expression after early intra-amniotic gene transfer using lentiviral vectors. Gene Therapy 2009, 17: 61-71. PMID: 19727133, DOI: 10.1038/gt.2009.115.Peer-Reviewed Original ResearchConceptsGene transferAccessible stem cell populationGene expressionDevelopmental stagesEarly gestational time pointsGene expression correspondsStem cell populationGreen fluorescent protein expressionTissue expressionCell populationsFluorescent protein expressionEarly somite stagesEpithelial cellsGerm layersLentiviral vectorsOnly epithelial cellsDevelopmental diseasesNeural ectodermGFP expressionSomite stageEfficient gene transferExpression correspondsProtein expressionEctodermGestational time points
1999
Egr-1 Mediates Extracellular Matrix-driven Transcription of Membrane Type 1 Matrix Metalloproteinase in Endothelium*
Haas T, Stitelman D, Davis S, Apte S, Madri J. Egr-1 Mediates Extracellular Matrix-driven Transcription of Membrane Type 1 Matrix Metalloproteinase in Endothelium*. Journal Of Biological Chemistry 1999, 274: 22679-22685. PMID: 10428849, DOI: 10.1074/jbc.274.32.22679.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsBase SequenceCloning, MolecularDNA-Binding ProteinsEarly Growth Response Protein 1Endothelium, VascularExtracellular MatrixGene Expression Regulation, EnzymologicHalf-LifeImmediate-Early ProteinsMatrix Metalloproteinase 14Matrix Metalloproteinases, Membrane-AssociatedMetalloendopeptidasesMiceMolecular Sequence DataNeovascularization, PhysiologicProtein BindingRatsRNA, MessengerSp1 Transcription FactorTranscription FactorsTranscription, GeneticUp-RegulationConceptsMembrane type 1 matrix metalloproteinaseEgr-1MT1-MMPTranscription factor Egr-1Number of proteinsExtracellular matrix environmentEnhanced transcriptional activityEndothelial cellsTranscriptional activityPromoter correlatesIncreased transcriptionCellular invasionInvasive phenotypeMatrix metalloproteinaseTranscriptionMatrix environmentMatrix metalloproteinase activityMetalloproteinase activityCellsMatrix metalloproteinasesInvasionIncrease productionAngiogenesisMetalloproteinaseProtein