2022
Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells
Luks VL, Mandl H, DiRito J, Barone C, Freedman-Weiss MR, Ricciardi AS, Tietjen GG, Egan ME, Saltzman WM, Stitelman DH. Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells. PLOS ONE 2022, 17: e0266218. PMID: 35385514, PMCID: PMC8986008, DOI: 10.1371/journal.pone.0266218.Peer-Reviewed Original ResearchConceptsTarget-specific antibodiesNanoparticle uptakeSurface conjugationNanoparticle surface modificationSurface of nanoparticlesCellular uptakeSite-specific geneSpecific cellular bindingNanoparticlesIntracellular deliveryEditing reagentsBronchial epithelial cellsSurface modificationCellular targetingCystic fibrosisTherapeutic agentsEpithelial cellsParticle uptakeFeasible strategyGenetic diseasesFirst demonstrationHuman bronchial epithelial cellsKinetics of antibodiesCellular bindingAppropriate antibodiesIntra-amniotic Injection of Poly(lactic-co-glycolic Acid) Microparticles Loaded with Growth Factor: Effect on Tissue Coverage and Cellular Apoptosis in the Rat Model of Myelomeningocele.
Maassel NL, Wu DH, Yung NK, Bauer-Pisani T, Elizabeth Guerra M, Ullrich SJ, Mark Saltzman W, Stitelman DH. Intra-amniotic Injection of Poly(lactic-co-glycolic Acid) Microparticles Loaded with Growth Factor: Effect on Tissue Coverage and Cellular Apoptosis in the Rat Model of Myelomeningocele. Journal Of The American College Of Surgeons 2022, 234: 1010-1019. PMID: 35703790, DOI: 10.1097/xcs.0000000000000156.Peer-Reviewed Original ResearchConceptsSoft tissue coverageIntra-amniotic injectionFibroblast growth factorTissue coverageRat modelMMC defectsGrowth factorCellular apoptosisGestational day 17Spinal cord cellsSitu cell deathCell deathWild-type controlsHistologic evidenceTreatment optionsLifelong morbidityUtero treatmentNeurologic disordersRat damsCord cellsMyelomeningoceleDay 17Invasive platformIncidenceInjection
2021
Diffusion weighted imaging as a biomarker of retinoic acid induced myelomeningocele
Maassel N, Farrelly J, Coman D, Freedman-Weiss M, Ahle S, Ullrich S, Yung N, Hyder F, Stitelman D. Diffusion weighted imaging as a biomarker of retinoic acid induced myelomeningocele. PLOS ONE 2021, 16: e0253583. PMID: 34191842, PMCID: PMC8244849, DOI: 10.1371/journal.pone.0253583.Peer-Reviewed Original ResearchConceptsMagnetic resonance imaging techniquesSpinal cordRat fetusesRetinoic acidCommon congenital anomalyComplementary imaging biomarkerLack of skinNeural tube defectsTrans retinoic acidBony coveringRat damsCongenital anomaliesRat modelExperimental therapiesHistopathologic analysisSevere formBrain volumeMyelomeningoceleImaging biomarkersDisease severityMMC defectsTube defectsIncomplete closureFetusesFractional anisotropy (FA) mapsEngineering alginate microparticles for optimized accumulation in fetal rat myelomeningocele
Freedman-Weiss MR, Wu D, Maassel N, Ullrich SJ, Ahle SL, Roberts K, Farrelly JS, Buzzelli G, Saltzman WM, Stitelman DH. Engineering alginate microparticles for optimized accumulation in fetal rat myelomeningocele. Journal Of Pediatric Surgery 2021, 57: 544-550. PMID: 33933264, DOI: 10.1016/j.jpedsurg.2021.03.060.Peer-Reviewed Original ResearchNanoparticles 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
Alginate microparticles loaded with basic fibroblast growth factor induce tissue coverage in a rat model of myelomeningocele
Farrelly JS, Bianchi AH, Ricciardi AS, Buzzelli GL, Ahle SL, Freedman-Weiss MR, Luks VL, Saltzman WM, Stitelman DH. Alginate microparticles loaded with basic fibroblast growth factor induce tissue coverage in a rat model of myelomeningocele. Journal Of Pediatric Surgery 2018, 54: 80-85. PMID: 30414695, DOI: 10.1016/j.jpedsurg.2018.10.031.Peer-Reviewed Original ResearchConceptsSoft tissue coverageTissue coverageRat modelTime-dated pregnant ratsFree bFGFGrowth factorInjection of microparticlesBasic fibroblast growth factorFibroblast growth factorPregnant ratsPrenatal treatmentIntraamniotic injectionFetal myelomeningoceleMyelomeningoceleMMC coverageMMC defectsRetinoic acidBFGFIn 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