Christina Barone
ClinicalTrials.gov CoordinatorCards
About
Research
Publications
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
Next generation triplex-forming PNAs for site-specific genome editing of the F508del CFTR mutation
Gupta A, Barone C, Quijano E, Piotrowski-Daspit A, Perera J, Riccardi A, Jamali H, Turchick A, Zao W, Saltzman W, Glazer P, Egan M. Next generation triplex-forming PNAs for site-specific genome editing of the F508del CFTR mutation. Journal Of Cystic Fibrosis 2024, 24: 142-148. PMID: 39107154, PMCID: PMC11788067, DOI: 10.1016/j.jcf.2024.07.009.Peer-Reviewed Original ResearchCystic fibrosis transmembrane conductance regulatorCystic fibrosis transmembrane conductance regulator geneF508del-CFTR mutationPeptide nucleic acidCFBE cellsBronchial epithelial cellsCystic fibrosisTriplex-forming peptide nucleic acidsDonor DNACFTR mutationsEpithelial cellsCFTR functionMutations associated with genetic diseasesPrimary nasal epithelial cellsAnalysis of genomic DNAGenetic diseasesIncreased CFTR functionDevelopment of peptide nucleic acidsImprove CFTR functionTransmembrane conductance regulatorAutosomal recessive genetic diseaseNasal epithelial cellsAir-liquid interfaceCystic fibrosis bronchial epithelial cellsHuman bronchial epithelial cells
2022
In vivo correction of cystic fibrosis mediated by PNA nanoparticles
Piotrowski-Daspit AS, Barone C, Lin CY, Deng Y, Wu D, Binns TC, Xu E, Ricciardi AS, Putman R, Garrison A, Nguyen R, Gupta A, Fan R, Glazer PM, Saltzman WM, Egan ME. In vivo correction of cystic fibrosis mediated by PNA nanoparticles. Science Advances 2022, 8: eabo0522. PMID: 36197984, PMCID: PMC9534507, DOI: 10.1126/sciadv.abo0522.Peer-Reviewed Original ResearchCystic fibrosisF508del miceIntravenous deliveryPrimary nasal epithelial cellsMultiple organ dysfunctionNasal epithelial cellsUssing chamber assaysOrgan dysfunctionF508del cystic fibrosisVivo treatmentGI tissuesCF transmembrane conductance regulator (CFTR) geneChamber assaySystemic deliveryEpithelial cellsCF-causing mutationsFibrosisCFTR functionMiceTransmembrane conductance regulator geneTarget effectsAir-liquid interfaceDeliveryPartial gainViable optionSurface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells
Luks VL, Mandl H, DiRito J, Barone C, Freedman-Weiss MR, Ricciardi AS, Tietjen GG, Egan ME, Saltzman WM, Stitelman DH. Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells. PLOS ONE 2022, 17: e0266218. PMID: 35385514, PMCID: PMC8986008, DOI: 10.1371/journal.pone.0266218.Peer-Reviewed Original ResearchConceptsTarget-specific antibodiesNanoparticle uptakeSurface conjugationNanoparticle surface modificationSurface of nanoparticlesCellular uptakeSite-specific geneSpecific cellular bindingNanoparticlesIntracellular deliveryEditing reagentsBronchial epithelial cellsSurface modificationCellular targetingCystic fibrosisTherapeutic agentsEpithelial cellsParticle uptakeFeasible strategyGenetic diseasesFirst demonstrationHuman bronchial epithelial cellsKinetics of antibodiesCellular bindingAppropriate antibodies
2021
582: In vivo nanoparticle-mediated therapeutic nucleic acid delivery for CF treatment
Piotrowski-Daspit A, Bracaglia L, Barone C, Nguyen R, Glazer P, Egan M, Saltzman W. 582: In vivo nanoparticle-mediated therapeutic nucleic acid delivery for CF treatment. Journal Of Cystic Fibrosis 2021, 20: s277. DOI: 10.1016/s1569-1993(21)02005-1.Peer-Reviewed Original Research
2016
Loss of Cystic Fibrosis Transmembrane Regulator Impairs Intestinal Oxalate Secretion
Knauf F, Thomson RB, Heneghan JF, Jiang Z, Adebamiro A, Thomson CL, Barone C, Asplin JR, Egan ME, Alper SL, Aronson PS. Loss of Cystic Fibrosis Transmembrane Regulator Impairs Intestinal Oxalate Secretion. Journal Of The American Society Of Nephrology 2016, 28: 242-249. PMID: 27313231, PMCID: PMC5198290, DOI: 10.1681/asn.2016030279.Peer-Reviewed Original ResearchConceptsIntestinal oxalate secretionWild-type miceCystic fibrosisIntestinal tissueOxalate secretionIncidence of hyperoxaluriaCalcium oxalate stone formationNet intestinal absorptionOxalate stone formationCoexpression of CFTRIntestinal transport processesWestern blot analysisOxalate absorptionMouse modelIntestinal absorptionGlucose absorptionUssing chambersStone formationFibrosisMiceSecretionReduced expressionCystic fibrosis transmembrane conductance regulator (CFTR) geneHyperoxaluriaPatientsMice lacking myosin IXb, an inflammatory bowel disease susceptibility gene, have impaired intestinal barrier function and superficial ulceration in the ileum
Hegan PS, Chandhoke SK, Barone C, Egan M, Bähler M, Mooseker MS. Mice lacking myosin IXb, an inflammatory bowel disease susceptibility gene, have impaired intestinal barrier function and superficial ulceration in the ileum. Cytoskeleton 2016, 73: 163-179. PMID: 26972322, DOI: 10.1002/cm.21292.Peer-Reviewed Original ResearchConceptsInflammatory bowel diseaseIntestinal barrier functionBarrier functionSuperficial ulcerationKO miceFeatures of IBDDextran sulfate sodium-induced colitisIntestinal mucosal damageMucosal barrier functionSodium-induced colitisFecal occult bloodIntestinal epithelial cell apoptosisInflammatory bowel disease susceptibility genesEpithelial cell apoptosisSusceptibility genesMyosin IXbTransepithelial electrical resistanceNeutrophil infiltrationMucosal damageBowel diseaseOccult bloodSurface ulcerationDisease onsetIntestinal mucosaFocal lesionsIncreased susceptibility of Cftr−/− mice to LPS-induced lung remodeling
Bruscia E, Zhang P, Barone C, Scholte BJ, Homer R, Krause D, Egan ME. Increased susceptibility of Cftr−/− mice to LPS-induced lung remodeling. American Journal Of Physiology - Lung Cellular And Molecular Physiology 2016, 310: l711-l719. PMID: 26851259, PMCID: PMC4836110, DOI: 10.1152/ajplung.00284.2015.Peer-Reviewed Original ResearchConceptsLung pathologyCF miceImmune responseWT miceChronic inflammationCystic fibrosisAbnormal immune responseChronic pulmonary infectionPersistent immune responseWild-type littermatesCF mouse modelsPseudomonas aeruginosa lipopolysaccharideCF lung pathologyPulmonary infectionChronic administrationLPS exposurePersistent inflammationLung remodelingWT littermatesLung tissueOverall pathologyMouse modelInflammationChronic exposureBacterial products