2024
Prenatal Therapy for Congenital Diaphragmatic Hernia and Myelomeningocele: Advances in Particle-Based Delivery
Rivero R, Stitelman D. Prenatal Therapy for Congenital Diaphragmatic Hernia and Myelomeningocele: Advances in Particle-Based Delivery. Current Stem Cell Reports 2024, 10: 51-57. DOI: 10.1007/s40778-024-00237-8.Peer-Reviewed Original ResearchCongenital diaphragmatic herniaDiaphragmatic herniaAnimal modelsIntra-amniotic injectionLong-term functional outcomeParticle-based deliveryFetal therapyPrenatal therapySpinal defectsTarget specific tissuesCo-morbiditiesCongenital diseaseFunctional outcomesTreatment deliveryStructural diseaseClinical translationMyelomeningoceleTherapyHerniaSpecific tissuesTissueDiseaseDeliveryPrenatallyParticle delivery
2023
In utero delivery of miRNA induces epigenetic alterations and corrects pulmonary pathology in congenital diaphragmatic hernia
Ullrich S, Yung N, Bauer-Pisani T, Maassel N, Guerra M, Freedman-Weiss M, Ahle S, Ricciardi A, Sauler M, Saltzman W, Piotrowski-Daspit A, Stitelman D. In utero delivery of miRNA induces epigenetic alterations and corrects pulmonary pathology in congenital diaphragmatic hernia. Molecular Therapy - Nucleic Acids 2023, 32: 594-602. PMID: 37200861, PMCID: PMC10185702, DOI: 10.1016/j.omtn.2023.04.018.Peer-Reviewed Original ResearchCongenital diaphragmatic herniaTGF-β pathwayDiaphragmatic herniaFetal ratsPulmonary vascular remodelingLung function resultsGestational day 18Pre-clinical modelsDifferent gestational timesCritical illnessPulmonary pathologyLung growthLung sizeRat modelFetal casesLung morphologyVascular remodelingMicroRNA-200bVein injectionFetal diseaseFirst breathDay 18Lung developmentGestational timeInvasive fashion
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