2025
Prevalence and Phenotype of Food Allergy in Children with Cystic Fibrosis
Nguyen H, Shabanova V, McCollum S, Polk B, Lee T, Whittington K, Cunha P, Bruscia E, Egan M, Emmenegger M, Leeds S. Prevalence and Phenotype of Food Allergy in Children with Cystic Fibrosis. Annals Of Allergy Asthma & Immunology 2025 PMID: 40550267, DOI: 10.1016/j.anai.2025.06.024.Peer-Reviewed Original ResearchCystic fibrosisCF patientsFood allergyUS pediatric populationElectronic medical recordsPediatric populationPhenotypes of food allergyRetrospective pediatric cohort studyPrevalence of FAFA prevalenceCystic Fibrosis Foundation RegistryMale CF patientsUnited StatesPediatric CF patientsPancreatic enzyme replacementPediatric cohort studyIgE-mediated FACohort studyEstimated prevalenceInclusion criteriaMedical informationDevelopment of FAMedical recordsFA diagnosisPrevalenceSystemic 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 systemENaC contributes to macrophage dysfunction in cystic fibrosis
Moran J, Pugh C, Brown N, Thomas A, Zhang S, McCauley E, Cephas A, Shrestha C, Partida-Sanchez S, Bai S, Bruscia E, Kopp B. ENaC contributes to macrophage dysfunction in cystic fibrosis. American Journal Of Physiology - Lung Cellular And Molecular Physiology 2025, 329: l61-l69. PMID: 40454714, PMCID: PMC12181047, DOI: 10.1152/ajplung.00009.2025.Peer-Reviewed Original ResearchConceptsCystic fibrosis transmembrane conductance regulatorCystic fibrosis transmembrane conductance regulator modulatorsMonocyte-derived macrophagesEpithelial sodium channelTransmembrane conductance regulatorCystic fibrosisImmune cellsProinflammatory cytokine productionENaC modulationENaC expressionConductance regulatorCystic fibrosis transmembrane conductance regulator inhibitionCytokine productionSodium channelsCF airway epithelial cellsReduced proinflammatory cytokine productionDecreased proinflammatory cytokine productionSodium channel expressionInfection controlTherapeutic targetAirway epithelial cellsIon channel dysfunctionReactive oxygen speciesIon channelsCFTR expressionEPS4.09The gut's role in pulmonary and extrapulmonary manifestation of cystic fibrosis
Cigana C, Delmonte G, Faccoetti A, Veschetti L, Gianferro F, Neri A, Fiorotto R, Livraghi-Butrico A, Massimino L, Ungaro F, Bragonzi A. EPS4.09The gut's role in pulmonary and extrapulmonary manifestation of cystic fibrosis. Journal Of Cystic Fibrosis 2025, 24: s52. DOI: 10.1016/j.jcf.2025.03.626.Peer-Reviewed Original ResearchWT miceCystic fibrosisHO miceDistal organsCollaborative CrossGenetically diverse Collaborative CrossInflammatory responseBarrier integrityManifestations of cystic fibrosisMouse models of CFEnteric bacteriaIncreased gut permeabilityCFTR modulator treatmentModel of CFEnhanced defense responsesGut barrier integrityImmune response pathwaysHigher bacterial loadsExtrapulmonary manifestationsSystemic inflammationChronic infectionInflammatory profileGut permeabilityGenomic toolsMicrobiological cultureWS16.05Impact of elexacaftor/tezacaftor/ivacaftor on glucose tolerance in adolescents with cystic fibrosis. Data from the MODUL-CF study
Bonnel A, Galderisi A, Weiss L, Sermet-Gaudelus I, Besancon A, Letierce A, Sahki D, Group M. WS16.05Impact of elexacaftor/tezacaftor/ivacaftor on glucose tolerance in adolescents with cystic fibrosis. Data from the MODUL-CF study. Journal Of Cystic Fibrosis 2025, 24: s33. DOI: 10.1016/j.jcf.2025.03.586.Peer-Reviewed Original ResearchOral glucose tolerance testAbnormal glucose toleranceNormal glucose toleranceCF-related diabetesBaseline oral glucose tolerance testAbnormal glucose tolerance groupCystic fibrosisGlucose toleranceNormal glucose tolerance groupEffective CFTR modulatorsOGTT glucoseBMI z-scoreYear of treatmentImpaired fasting glucoseImpaired glucose toleranceGlucose tolerance testForced expiratory volumeCFTR modulatorsPulmonary infectionDecrease recurrenceBaseline characteristicsRelated diabetesFasting glucoseTolerance testInsulin treatmentPersonalized Bacteriophage Therapy Reduces Pseudomonas Aeruginosa Biofilm Formation and Elicits an Anti-inflammatory Effect in Cystic Fibrosis
Borges W, Chan B, Kazmierczak B, Sun Y, Turner P, Koff J, Stanley G. Personalized Bacteriophage Therapy Reduces Pseudomonas Aeruginosa Biofilm Formation and Elicits an Anti-inflammatory Effect in Cystic Fibrosis. American Journal Of Respiratory And Critical Care Medicine 2025, 211: a4554-a4554. DOI: 10.1164/ajrccm.2025.211.abstracts.a4554.Peer-Reviewed Original ResearchPersonalized inhaled bacteriophage therapy for treatment of multidrug-resistant Pseudomonas aeruginosa in cystic fibrosis
Chan B, Stanley G, Kortright K, Vill A, Modak M, Ott I, Sun Y, Würstle S, Grun C, Kazmierczak B, Rajagopalan G, Harris Z, Britto C, Stewart J, Talwalkar J, Appell C, Chaudary N, Jagpal S, Jain R, Kanu A, Quon B, Reynolds J, Teneback C, Mai Q, Shabanova V, Turner P, Koff J. Personalized inhaled bacteriophage therapy for treatment of multidrug-resistant Pseudomonas aeruginosa in cystic fibrosis. Nature Medicine 2025, 31: 1494-1501. PMID: 40301561, PMCID: PMC12092284, DOI: 10.1038/s41591-025-03678-8.Peer-Reviewed Original ResearchConceptsPhage therapyCystic fibrosisEvidence of trade-offsTreated with phagesEvolutionary trade-offClinical impact of antimicrobial resistanceMultidrug-resistant Pseudomonas aeruginosaBacterial virulenceAntimicrobial resistance crisisLytic virusesDecrease antibiotic resistanceSputum microbiomePhageBacteriophage therapyImpact of antimicrobial resistanceAntibiotic resistanceResistance crisisStandard antibioticsAntimicrobial resistanceSputum densityMultidrug resistancePulmonary infectionCompassionate basisClinical courseMedian ageEffect of Elexacaftor/Tezacaftor/Ivacaftor on Glucose Tolerance in Adolescents With Cystic Fibrosis
Galderisi A, Weiss L, Besançon A, Stremler N, Reix P, Wizla N, Lustre A, Rames C, Tatopoulos A, Perisson C, Dalphin M, Troussier F, Houdouin V, Bessaci K, Cosson L, Gabsi A, Corvol H, Deneuville E, Storni V, Ramel S, Bui S, Heraud M, Remus N, Huet F, Scalbert M, Mely L, Gachelin E, Giannantonio M, Letierce A, Sahki D, Marguet C, Bonnel A, Sermet-Gaudelus I. Effect of Elexacaftor/Tezacaftor/Ivacaftor on Glucose Tolerance in Adolescents With Cystic Fibrosis. The Journal Of Clinical Endocrinology & Metabolism 2025, dgaf099. PMID: 39977216, DOI: 10.1210/clinem/dgaf099.Peer-Reviewed Original ResearchOral glucose tolerance testBaseline oral glucose tolerance testAbnormal glucose toleranceNormal glucose toleranceAbnormal glucose tolerance groupCystic fibrosisGlucose toleranceNormal glucose tolerance groupEffective CFTR modulatorsCF-related diabetesInsulin secretionImpaired fasting glucoseGlucose tolerance testImpaired glucose toleranceForced expiratory volumeBMIz-scoreCFTR modulatorsBaseline characteristicsRelated diabetesFasting glucoseTolerance testTherapeutic strategiesInsulin treatmentExpiratory volumeGlucose levelsCFTR dictates monocyte adhesion by facilitating integrin clustering but not activation
Younis D, Marosvari M, Liu W, Pulikkot S, Cao Z, Zhou B, Vella A, McArdle S, Hu L, Chen Y, Gan W, Yu J, Bruscia E, Fan Z. CFTR dictates monocyte adhesion by facilitating integrin clustering but not activation. Proceedings Of The National Academy Of Sciences Of The United States Of America 2025, 122: e2412717122. PMID: 39813254, PMCID: PMC11760921, DOI: 10.1073/pnas.2412717122.Peer-Reviewed Original ResearchConceptsIntegrin clusteringCF transmembrane conductance regulatorCystic fibrosisAdhesion defectsPathogenesis of cystic fibrosisClinically relevant disease modelsMembrane recruitmentTransmembrane conductance regulatorIntegrin activationTherapeutic strategy designRelevant disease modelsIntegrinCF monocytesCell adhesionMonocyte dysfunctionPatients' monocytesTissue infectionsConductance regulatorSuperresolution microscopyCortex formationLeukocyte-dependent inflammationInflammatory pathogenesisLeukocyte adhesionMonocytesInflammationCFTR High Expresser BEST4+ cells are pH-sensing neuropod cells: new implications for intestinal physiology and Cystic Fibrosis disease
dos Reis D, Jin J, Santos A, Nogueira C, Zagoren E, Donnelley M, Parsons D, Cmielewski P, Reyne N, Mc Carron A, Smith Z, Sumigray K, Ameen N. CFTR High Expresser BEST4+ cells are pH-sensing neuropod cells: new implications for intestinal physiology and Cystic Fibrosis disease. Physiology 2025, 40: 1127. DOI: 10.1152/physiol.2025.40.s1.1127.Peer-Reviewed Original ResearchCHE cellsGuanylyl cyclase-CNeuropod cellsApical domainRat jejunumHuman intestineSingle-cell RNA sequencingCystic fibrosisLuminal pH regulationSubpopulation of epithelial cellsLong basal processesScRNA-seqRNA sequencingAcid-sensing receptorsWild-type animalsCystic fibrosis diseaseIncreased abundanceHigh-expressing cellsProtein immunolocalizationIntestinal physiologyMorphological coreWild-typeBasal processesHCO3- secretionCFTR
2024
CCR2+ monocytes are dispensable to resolve acute pulmonary Pseudomonas aeruginosa infections in WT and cystic fibrosis mice
Öz H, Braga C, Gudneppanavar R, Di Pietro C, Huang P, Zhang P, Krause D, Egan M, Murray T, Bruscia E. CCR2+ monocytes are dispensable to resolve acute pulmonary Pseudomonas aeruginosa infections in WT and cystic fibrosis mice. Journal Of Leukocyte Biology 2024, 117: qiae218. PMID: 39365279, PMCID: PMC11953069, DOI: 10.1093/jleuko/qiae218.Peer-Reviewed Original ResearchLung tissue damageCystic fibrosisTissue damageMonocyte recruitmentImmune responsePulmonary Pseudomonas aeruginosa infectionHyper-inflammatory immune responseCystic fibrosis micePropagate tissue damagePseudomonas aeruginosaLungs of patientsChronic neutrophilic inflammationImmunological response to infectionHost immune responseMonocyte-derived macrophagesTarget monocyte recruitmentSite of injuryResponse to infectionCFTR modulatorsPA infectionChronic inflammatory disease conditionsReduced bactericidal activityAdjunctive therapyClinical outcomesEradicate infection219 CFTR dysfunction shapes airway immune cell compositions contributing to lung pathogenesis in children with cystic fibrosis
Kizilirmak T, Yin H, Garrison A, Browne J, Bruscia E, Egan M, Britto C. 219 CFTR dysfunction shapes airway immune cell compositions contributing to lung pathogenesis in children with cystic fibrosis. Journal Of Cystic Fibrosis 2024, 23: s119. DOI: 10.1016/s1569-1993(24)01059-2.Peer-Reviewed Original Research689 Triple CFTR modulator combination improves glucose tolerance in adolescents with cystic fibrosis: data from the French observational pediatric study MODUL-CF
Weiss L, Galderisi A, Besançon A, Stremler N, Reix P, Wizla N, Rames C, Marguet C, Tatapoulos A, Perrisson C, Dalphin M, Troussier F, Houdouin V, Abely M, Cosson L, Gabsi A, Corvol H, Deneuville E, Sahki D, Bonnel A, Group M, Sermet-Gaudelus I. 689 Triple CFTR modulator combination improves glucose tolerance in adolescents with cystic fibrosis: data from the French observational pediatric study MODUL-CF. Journal Of Cystic Fibrosis 2024, 23: s386-s387. DOI: 10.1016/s1569-1993(24)01527-3.Peer-Reviewed Original ResearchCFTR modulator combinationsCystic fibrosisPathophysiology of Cystic Fibrosis Liver Disease
Kasper V, Assis D. Pathophysiology of Cystic Fibrosis Liver Disease. Pediatric Pulmonology 2024, 59: s98-s106. PMID: 39105342, DOI: 10.1002/ppul.26869.Peer-Reviewed Original ResearchConceptsComplication of cystic fibrosisCF liver diseaseHeterogeneity of clinical manifestationsManifestations of CFDisease-modifying factorsCFTR mutationsHepatobiliary complicationsBiliary stricturesBiliary cholestasisClinical manifestationsCystic fibrosisLiver diseaseTherapeutic approachesCF careDisease modelsDiseaseNext 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 cellsLocalization and function of humanized F508del-CFTR in mouse intestine following activation of serum glucocorticoid kinase 1 and Trikafta
Dastoor P, Muiler C, Garrison A, Egan M, Carlos Dos Reis D, Santos A, Ameen N. Localization and function of humanized F508del-CFTR in mouse intestine following activation of serum glucocorticoid kinase 1 and Trikafta. European Journal Of Pharmacology 2024, 978: 176771. PMID: 38925289, DOI: 10.1016/j.ejphar.2024.176771.Peer-Reviewed Original ResearchSerum glucocorticoid kinase 1Glucocorticoid kinase 1F508del-CFTRCystic fibrosisMouse modelF508del-CFTR miceCFTR-expressing epithelial cellsCF mouse modelsHumanized mouse modelTreatment of ratsIntestinal diseaseIntestinal segmentsKinase 1CFTR modulatorsCFTR mutationsCF patientsTrikaftaDex treatmentLung diseaseEfficacy of compoundsDelta F508CFTRCombined treatmentEpithelial cellsLoss of functionNeutralizing antibodies after nebulized phage therapy in cystic fibrosis patients
Bernabéu-Gimeno M, Pardo-Freire M, Chan B, Turner P, Gil-Brusola A, Pérez-Tarazona S, Carrasco-Hernández L, Quintana-Gallego E, Domingo-Calap P. Neutralizing antibodies after nebulized phage therapy in cystic fibrosis patients. Med 2024, 5: 1096-1111.e6. PMID: 38917792, DOI: 10.1016/j.medj.2024.05.017.Peer-Reviewed Original ResearchMulti-drug resistanceCystic fibrosis patientsPhage therapyClinical outcomesLung infectionFibrosis patientsCystic fibrosisPseudomonas aeruginosa lung infectionAnti-phage antibodiesImmune responseStandard-of-care antibioticsInvasive routes of administrationPhage-neutralizing antibodiesBacterial loadBacterial lung infectionsCystic Fibrosis FoundationNo adverse eventsRoute of administrationPhage preparationsEmergence of antibodiesPhage detectionAntibiotic susceptibilityPhageStaphylococcus aureusBacterial eradicationIs Lipid Metabolism of Value in Cancer Research and Treatment? Part II: Role of Specialized Pro-Resolving Mediators in Inflammation, Infections, and Cancer
Babar M, Nassar A, Nie X, Zhang T, He J, Yeung J, Norris P, Ogura H, Muldoon A, Chen L, Libreros S. Is Lipid Metabolism of Value in Cancer Research and Treatment? Part II: Role of Specialized Pro-Resolving Mediators in Inflammation, Infections, and Cancer. Metabolites 2024, 14: 314. PMID: 38921449, PMCID: PMC11205484, DOI: 10.3390/metabo14060314.Peer-Reviewed Original ResearchResolution of inflammationPro-resolving mediatorsTherapeutic effectOral squamous cell carcinomaT cell responsesSquamous cell carcinomaEicosapentaenoic acid derivativeExcessive neutrophil infiltrationDendritic cell migrationEndogenous lipid mediatorsMetastasis of cancer cellsPro-inflammatory cytokinesSpecialized pro-resolving mediatorsCell carcinomaTumor microenvironmentIschemia-related injuryLung inflammationTumor progressionNeutrophil infiltrationCell debris clearanceCystic fibrosisLung cancerAcute inflammationHerpes virusTherapeutic approachesStructure-based discovery of CFTR potentiators and inhibitors
Liu F, Kaplan A, Levring J, Einsiedel J, Tiedt S, Distler K, Omattage N, Kondratov I, Moroz Y, Pietz H, Irwin J, Gmeiner P, Shoichet B, Chen J. Structure-based discovery of CFTR potentiators and inhibitors. Cell 2024, 187: 3712-3725.e34. PMID: 38810646, PMCID: PMC11262615, DOI: 10.1016/j.cell.2024.04.046.Peer-Reviewed Original ResearchConceptsLarge-scale dockingIon channel drug discoveryStructure-based discoveryStructure-based optimizationMedicinal chemistryTested ligandsMolecular dockingSmall moleculesDrug discoveryCystic fibrosis transmembrane conductance regulatorMoleculesAllosteric siteIonsDockingLigandSecretory diarrheaCystic fibrosisCystic fibrosis transmembrane conductance regulator modulatorsChemistryTransmembrane conductance regulatorSuboptimal pharmacokineticsCFTR potentiatorsSynergistic effects of sulopenem in combination with cefuroxime or durlobactam against Mycobacterium abscessus
Dousa K, Shin E, Kurz S, Plummer M, Nantongo M, Bethel C, Taracila M, Nguyen D, Kreiswith B, Daley C, Remy K, Holland S, Bonomo R. Synergistic effects of sulopenem in combination with cefuroxime or durlobactam against Mycobacterium abscessus. MBio 2024, 15: e00609-24. PMID: 38742824, PMCID: PMC11237399, DOI: 10.1128/mbio.00609-24.Peer-Reviewed Original ResearchConceptsD,D-carboxypeptidaseMinimum inhibitory concentrationB-lactamPenicillin-binding-proteinCell wall synthesisL,D-transpeptidasesResistance to common antibioticsLowest minimum inhibitory concentrationWall synthesisCystic fibrosisD-carboxypeptidaseMass spectrometry analysisB-lactamaseThioester bondRate of treatment failureCell-based assaysMultiple antibioticsB-lactam ringsCommon antibioticsD-transpeptidasesB-lactamase inhibitorsCysteine residuesTime-kill studiesStructural lung diseaseMultidrug-resistant tuberculosis
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