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 disease