2025
Systemic in utero gene editing as a treatment for cystic fibrosis
Ricciardi A, Barone C, Putman R, Quijano E, Gupta A, Nguyen R, Mandl H, Piotrowski-Daspit A, Lopez-Giraldez F, Luks V, Freedman-Weiss M, Farrelly J, Ahle S, Lynn A, Glazer P, Saltzman W, Stitelman D, Egan M. Systemic in utero gene editing as a treatment for cystic fibrosis. Proceedings Of The National Academy Of Sciences Of The United States Of America 2025, 122: e2418731122. PMID: 40493185, PMCID: PMC12184489, DOI: 10.1073/pnas.2418731122.Peer-Reviewed Original ResearchConceptsUtero gene editingCystic fibrosisCF transmembrane conductance regulatorTreat CF patientsTransmembrane conductance regulatorWild-type miceIrreversible organ damageNormal organ developmentTreat monogenic diseasesCFTR activityCF patientsConductance regulatorDisease-causing genesMultiorgan diseaseDisease improvementOrgan damageGene editingMonogenic diseasesMutation correctionPolymeric nanoparticlesGastrointestinal tissuesDiseaseBirthFibrosisReproductive system
2024
Biodistribution of Polymeric Nanoparticles following in utero Delivery to a Nonhuman 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 Nonhuman Primate. Biomedicine Hub 2024, 10: 543138. PMID: 39845408, PMCID: PMC11753793, 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 nanoparticlesDiseaseBirthMiceDeliveryBiodistributionEnhancing in vivo cell and tissue targeting by modulation of polymer nanoparticles and macrophage decoys
Piotrowski-Daspit A, Bracaglia L, Eaton D, Richfield O, Binns T, Albert C, Gould J, Mortlock R, Egan M, Pober J, Saltzman W. Enhancing in vivo cell and tissue targeting by modulation of polymer nanoparticles and macrophage decoys. Nature Communications 2024, 15: 4247. PMID: 38762483, PMCID: PMC11102454, DOI: 10.1038/s41467-024-48442-7.Peer-Reviewed Original ResearchConceptsPoly(amine-co-esterPolymer nanoparticlesDelivery of nucleic acid therapeuticsCell-type tropismTissue tropismNucleic acid delivery vehiclesIn vivo deliveryIn vivo efficacyCirculation half-lifeNucleic acid therapeuticsVehicle characteristicsTunable propertiesBiodistribution assessmentPhysiological fatePolymer chemistrySurface propertiesPharmacokinetic modelTissue targetingNanoparticlesDistribution modifiersPolymeric nanoparticlesTropismPolymerDelivery vehiclesHalf-life
2021
Comprehensive review on ultrasound-responsive theranostic nanomaterials: mechanisms, structures and medical applications
Fateh S, Moradi L, Kohan E, Hamblin M, Dezfuli A. Comprehensive review on ultrasound-responsive theranostic nanomaterials: mechanisms, structures and medical applications. Beilstein Journal Of Nanotechnology 2021, 12: 808-862. PMID: 34476167, PMCID: PMC8372309, DOI: 10.3762/bjnano.12.64.Peer-Reviewed Original ResearchTheranostic nanomaterialsCell deathReactive oxygen species generationClinical imaging modalitiesOxygen species generationTherapeutic effectImaging modalitiesMesoporous silica nanoparticlesPolymeric nanoparticlesRelease of encapsulated drugsAcoustic radiation forceTitania nanostructuresSpecies generationCarbon nanostructuresLocal thermal effectsUltrasoundDroplet vaporizationSilica nanoparticlesAcoustic droplet vaporizationCavitation microbubblesDeathTheranosticsThermal effectsNanoparticlesNanomaterials
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
2017
Degradable bioadhesive nanoparticles for prolonged intravaginal delivery and retention of elvitegravir
Mohideen M, Quijano E, Song E, Deng Y, Panse G, Zhang W, Clark MR, Saltzman WM. Degradable bioadhesive nanoparticles for prolonged intravaginal delivery and retention of elvitegravir. Biomaterials 2017, 144: 144-154. PMID: 28829952, PMCID: PMC5581224, DOI: 10.1016/j.biomaterials.2017.08.029.Peer-Reviewed Original ResearchConceptsPolymer carrierPolymeric nanoparticlesIntravaginal deliveryBioadhesive nanoparticlesNanoparticlesHuman immunodeficiency virusPromising strategyProtective mucus layerReproductive tractFormulation 1Residence timePolyEpithelial cell populationsSite of actionImmunodeficiency virusRetentionIntravaginal administrationVaginal lumenMucosal surfacesSustained protectionTopical applicationSTD epidemicsElvitegravirHormone cyclesMucus layer
2013
Highly penetrative, drug-loaded nanocarriers improve treatment of glioblastoma
Zhou J, Patel TR, Sirianni RW, Strohbehn G, Zheng MQ, Duong N, Schafbauer T, Huttner AJ, Huang Y, Carson RE, Zhang Y, Sullivan DJ, Piepmeier JM, Saltzman WM. Highly penetrative, drug-loaded nanocarriers improve treatment of glioblastoma. Proceedings Of The National Academy Of Sciences Of The United States Of America 2013, 110: 11751-11756. PMID: 23818631, PMCID: PMC3718184, DOI: 10.1073/pnas.1304504110.Peer-Reviewed Original Research
2011
Polymeric nanoparticles for drug delivery to the central nervous system
Patel T, Zhou J, Piepmeier JM, Saltzman WM. Polymeric nanoparticles for drug delivery to the central nervous system. Advanced Drug Delivery Reviews 2011, 64: 701-705. PMID: 22210134, PMCID: PMC3323692, DOI: 10.1016/j.addr.2011.12.006.Peer-Reviewed Original Research
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