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 deliveryThe Technical and Ethical Framework of Fetal Therapy: Past and Current Advances
Lynn A, Glazer P, Saltzman W, Stitelman D. The Technical and Ethical Framework of Fetal Therapy: Past and Current Advances. Current Stem Cell Reports 2024, 10: 30-36. DOI: 10.1007/s40778-024-00235-w.Peer-Reviewed Original ResearchFetal drug deliverySuccess of nanomedicineDrug deliveryPharmacological drug treatmentsMinimally invasive strategyFetal therapyFetal applicationsClinical studiesCongenital diseaseInvasive strategyDrug treatmentClinical translationClinical practiceTherapyMedical interventionsDeliveryFetalNanomedicineBirthDiseaseFindingsAClinicians
2021
A narrative review of in utero gene therapy: advances, challenges, and future considerations
Yung NK, Maassel NL, Ullrich SJ, Ricciardi AS, Stitelman DH. A narrative review of in utero gene therapy: advances, challenges, and future considerations. Translational Pediatrics 2021, 10: 1486496-1481496. PMID: 34189107, PMCID: PMC8192997, DOI: 10.21037/tp-20-89.Peer-Reviewed Original ResearchHighest effective doseGene therapyPathogenesis increasesLysosomal storage disorderImmune tolerancePostnatal treatmentTreatment optionsClinical trialsAnimal modelsCystic fibrosisFetal diagnosisNarrative reviewTranslational gapEffective doseUtero gene therapyPhysiologic phenomenonStorage disorderDiseaseNext-generation sequencingIUGTTechnologic advancementsCandidate diseaseClinical realmTherapyPotential diseaseNanoparticles 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
2020
Minimally Invasive Fetal Therapy for Myelomeningocele
Freedman-Weiss M, Stitelman D. Minimally Invasive Fetal Therapy for Myelomeningocele. Current Stem Cell Reports 2020, 6: 1-5. DOI: 10.1007/s40778-020-00167-1.Peer-Reviewed Original ResearchNon-invasive therapeutic approachInvasive fetal therapyPostnatal surgical repairDevastating clinical manifestationsTissue engineeringScaffold-based tissue engineeringMedical managementSurgical repairCell-based therapiesClinical manifestationsFetal stem cellsCurrent therapiesPatient outcomesSpinal cordFetal therapyFetal surgeryTherapeutic approachesTherapyInvasive wayTranslational scienceStem cellsReview detailsParticle deliveryNovel scientific researchParticle injection