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 nanoparticlesDiseaseBirthMiceDeliveryBiodistribution
2018
In 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