2024
Biodistribution of Polymeric Nanoparticles following In-Utero Delivery to a Non-Human Primate
Eaton D, Lynn A, Surprenant J, Deschenes E, Guerra M, Rivero R, Yung N, O'Connor M, Glazer P, Bahtiyar M, Saltzman W, Stitelman D. Biodistribution of Polymeric Nanoparticles following In-Utero Delivery to a Non-Human Primate. Biomedicine Hub 2024, 1-16. DOI: 10.1159/000543138.Peer-Reviewed Original ResearchMonogenic diseasesNon-human primatesBiodistribution of polymeric nanoparticlesPreclinical mouse modelsStem cell populationFetal liverFetal malesSystemic deliveryFetal bonesAccumulation of NPIn-uteroMid-gestationNP accumulationBiodistribution studiesPolymeric NPsTherapeutic accessCell populationsMaternal tissuesZone of proliferationPolymeric nanoparticlesDiseaseBirthMiceDeliveryBiodistributionPrenatal 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