2025
CFTR 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 ResearchMeSH KeywordsCell AdhesionCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorHumansInflammationIntegrinsMonocytesMutationConceptsIntegrin 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 adhesionMonocytesInflammation
2024
Pathophysiology 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 ResearchMeSH KeywordsCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorHumansLiver DiseasesMutationConceptsComplication of cystic fibrosisCF liver diseaseHeterogeneity of clinical manifestationsManifestations of CFDisease-modifying factorsCFTR mutationsHepatobiliary complicationsBiliary stricturesBiliary cholestasisClinical manifestationsCystic fibrosisLiver diseaseTherapeutic approachesCF careDisease modelsDiseaseStructure-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 potentiatorsLytic bacteriophages induce the secretion of antiviral and proinflammatory cytokines from human respiratory epithelial cells
Zamora P, Reidy T, Armbruster C, Sun M, Van Tyne D, Turner P, Koff J, Bomberger J. Lytic bacteriophages induce the secretion of antiviral and proinflammatory cytokines from human respiratory epithelial cells. PLOS Biology 2024, 22: e3002566. PMID: 38652717, PMCID: PMC11037538, DOI: 10.1371/journal.pbio.3002566.Peer-Reviewed Original ResearchConceptsLytic phagesLytic bacteriophagesPhage therapyAirway epithelial cellsPseudomonas aeruginosa phagesEpithelial cellsMultidrug resistanceAirway epitheliumCystic fibrosisProinflammatory cytokinesHuman respiratory epithelial cellsPhage exposurePhage familiesMammalian cell responsesHuman airway epithelial cellsInternalized phageTreat multidrug-resistantPhageBacterial isolatesTranscriptional profilesRespiratory epithelial cellsHuman hostChronic respiratory disordersBacterial biofilmsBacteriophageNovel Approaches to Multidrug-Resistant Infections in Cystic Fibrosis
Murray T, Stanley G, Koff J. Novel Approaches to Multidrug-Resistant Infections in Cystic Fibrosis. Infectious Disease Clinics Of North America 2024, 38: 149-162. PMID: 38280761, DOI: 10.1016/j.idc.2023.12.002.Peer-Reviewed Original ResearchConceptsMultidrug-resistant organismsCystic fibrosis transmembrane conductance regulatorCystic fibrosisBeta-lactam/beta-lactamase combinationDevelopment of inhaled formulationsMethicillin-resistant Staphylococcus aureusTherapeutic approachesMultidrug-resistant organism infectionSystemic adverse eventsMultidrug-resistant infectionsTransmembrane conductance regulatorRespiratory tract infectionsCystic fibrosis patientsGram-negative organismsInnovative therapeutic approachesPulmonary infectionTract infectionsConductance regulatorBurkholderia sp.Multidrug resistanceAdverse eventsTreatment optionsAntibiotic resistanceFibrosis patientsOptimal dose
2023
Cystic fibrosis screening, evaluation, and management of hepatobiliary disease consensus recommendations
Sellers Z, Assis D, Paranjape S, Sathe M, Bodewes F, Bowen M, Cipolli M, Debray D, Green N, Hughan K, Hunt W, Leey J, Ling S, Morelli G, Peckham D, Pettit R, Philbrick A, Stoll J, Vavrina K, Allen S, Goodwin T, Hempstead S, Narkewicz M. Cystic fibrosis screening, evaluation, and management of hepatobiliary disease consensus recommendations. Hepatology 2023, 79: 1220-1238. PMID: 37934656, PMCID: PMC11020118, DOI: 10.1097/hep.0000000000000646.Peer-Reviewed Original ResearchConceptsAdvanced liver diseaseCystic fibrosisLiver diseaseHepatobiliary involvementHepatobiliary complicationsCF-associated liver diseasePubMed literature searchCystic fibrosis screeningScreening ultrasoundPortal hypertensionOutcome questionsLiver biopsyLiver failureMultilobular cirrhosisFibrosis screeningConsensus recommendationsFull-text reviewHepatobiliary diseaseCF FoundationLiterature searchThirty-oneCF outcomesMultidisciplinary committeeDiseaseComplicationsOpen-Label Trial of Amikacin Liposome Inhalation Suspension in Mycobacterium abscessus Lung Disease
Siegel S, Griffith D, Philley J, Brown-Elliott B, Brunton A, Sullivan P, Fuss C, Strnad L, Wallace R, Winthrop K. Open-Label Trial of Amikacin Liposome Inhalation Suspension in Mycobacterium abscessus Lung Disease. CHEST Journal 2023, 164: 846-859. PMID: 37419144, PMCID: PMC10645596, DOI: 10.1016/j.chest.2023.05.036.Peer-Reviewed Original ResearchConceptsAmikacin liposome inhalation suspensionAmikacin resistanceCulture conversionInhalation suspensionMycobacterium abscessus lung diseasePresence of macrolide resistanceEnd pointsMicrobiological end pointsMonthly sputum culturesSputum culture conversionTreatment-refractory diseaseOpen-label protocolSecondary end pointsOpen-label trialCohort of patientsCavitary diseaseSputum culturePretreatment isolatesM abscessusOpen-labelOral antimicrobialsParenteral antibioticsCompanion therapyMacrolide resistanceMultidrug therapyScreening strategies for glucose tolerance abnormalities and diabetes in people with cystic fibrosis
Weiss L, Reix P, Mosnier-Pudar H, Ronsin O, Beltrand J, Reynaud Q, Mely L, Burgel P, Stremler N, Rakotoarisoa L, Galderisi A, Perge K, Bendelac N, Abely M, Kessler L. Screening strategies for glucose tolerance abnormalities and diabetes in people with cystic fibrosis. Diabetes & Metabolism 2023, 49: 101444. PMID: 37030530, DOI: 10.1016/j.diabet.2023.101444.Peer-Reviewed Original ResearchMeSH KeywordsAdultBlood GlucoseChildComorbidityCystic FibrosisDiabetes MellitusGlucoseGlucose IntoleranceGlucose Tolerance TestHumansConceptsGlucose tolerance abnormalitiesCystic fibrosis-related diabetesTolerance abnormalitiesCystic fibrosisAnnual oral glucose tolerance testOral glucose tolerance testGlucose tolerance testLong asymptomatic periodStage of diabetesNew comorbiditiesAsymptomatic periodClinical profileTolerance testNew diagnostic toolsPathophysiological knowledgeDiabetesNutritional outcomesPatientsFibrosisAbnormalitiesScreening strategyLife expectancyDiagnostic toolScreening methodComorbiditiesThe effects of elexafactor/tezafactor/ivacaftor beyond the epithelium: spurring macrophages to fight infections.
Bruscia E. The effects of elexafactor/tezafactor/ivacaftor beyond the epithelium: spurring macrophages to fight infections. European Respiratory Journal 2023, 61: 2300216. PMID: 37003613, DOI: 10.1183/13993003.00216-2023.Peer-Reviewed Original ResearchMeSH KeywordsAminophenolsCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorEpitheliumHumansMacrophagesFuture therapies for cystic fibrosis
Allen L, Allen L, Carr S, Davies G, Downey D, Egan M, Forton J, Gray R, Haworth C, Horsley A, Smyth A, Southern K, Davies J. Future therapies for cystic fibrosis. Nature Communications 2023, 14: 693. PMID: 36755044, PMCID: PMC9907205, DOI: 10.1038/s41467-023-36244-2.Peer-Reviewed Original ResearchMeSH KeywordsCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorGenetic TherapyHumansMutationConceptsMutation-specific drugsCystic fibrosisSymptom-directed treatmentMultisystem clinical manifestationsCystic fibrosis therapyCystic fibrosis transmembrane conductance regulatorGenetic variantsClinical manifestationsFuture therapiesFibrosis therapyTranslational research collaborationsModulator drugsCFTR modulatorsSingle gene disordersHealth inequalitiesTherapyGene variantsImproved treatmentDrugsPatientsFibrosisFibrosis transmembrane conductance regulatorGene disordersTransmembrane conductance regulatorStrategy groupBacteriophage Therapy for Pan-Drug-Resistant Pseudomonas aeruginosa in Two Persons With Cystic Fibrosis
Hahn A, Sami I, Chaney H, Koumbourlis A, Del Valle Mojica C, Cochrane C, Chan B, Koff J. Bacteriophage Therapy for Pan-Drug-Resistant Pseudomonas aeruginosa in Two Persons With Cystic Fibrosis. Journal Of Investigative Medicine High Impact Case Reports 2023, 11: 23247096231188243. PMID: 37515541, PMCID: PMC10387758, DOI: 10.1177/23247096231188243.Peer-Reviewed Original ResearchMeSH KeywordsChildCystic FibrosisHumansLungPhage TherapyPseudomonas aeruginosaPseudomonas InfectionsConceptsPulmonary exacerbationsCF transmembrane conductance regulator geneTransmembrane conductance regulator geneChronic bacterial colonizationRecurrent lung infectionsStructural lung damagePediatric clinical trialsLung function declineLung inflammationTreatment modalitiesLung infectionCystic fibrosisLung damageClinical trialsTherapeutic strategiesAntibiotic resistanceBacterial colonizationMonogenic diseasesBacteriophage therapyRegulated genesViscous secretionsReduced survivalFunctional declineExacerbationBacteriophage
2022
Inhaled Bacteriophage Therapy for Multi-Drug Resistant Achromobacter.
Winzig F, Gandhi S, Lee A, Würstle S, Stanley G, Capuano I, Neuringer I, Koff J, Turner P, Chan B. Inhaled Bacteriophage Therapy for Multi-Drug Resistant Achromobacter. The Yale Journal Of Biology And Medicine 2022, 95: 413-427. PMID: 36568830, PMCID: PMC9765334.Peer-Reviewed Original ResearchConceptsCF patientsCystic fibrosisChronic pulmonary infectionGlobal public health threatBacterial lung infectionsChallenging clinical problemPublic health threatChronic bacterial lung infectionsPulmonary infectionRespiratory statusLung infectionClinical problemBacteriophage therapyInfectionAntimicrobial-resistant bacteriaTherapyHealth threatPhage therapyPatientsAMR infectionsResistant bacteriaLytic bacteriophagesPossible benefitsChemical antibioticsCurrent studyRecruited monocytes/macrophages drive pulmonary neutrophilic inflammation and irreversible lung tissue remodeling in cystic fibrosis
Öz H, Cheng E, Di Pietro C, Tebaldi T, Biancon G, Zeiss C, Zhang P, Huang P, Esquibies S, Britto C, Schupp J, Murray T, Halene S, Krause D, Egan M, Bruscia E. Recruited monocytes/macrophages drive pulmonary neutrophilic inflammation and irreversible lung tissue remodeling in cystic fibrosis. Cell Reports 2022, 41: 111797. PMID: 36516754, PMCID: PMC9833830, DOI: 10.1016/j.celrep.2022.111797.Peer-Reviewed Original ResearchConceptsC motif chemokine receptor 2Monocytes/macrophagesLung tissue damageCystic fibrosisTissue damageCF lungPulmonary neutrophilic inflammationPro-inflammatory environmentChemokine receptor 2CF lung diseaseNumber of monocytesSpecific therapeutic agentsGrowth factor βCF transmembrane conductance regulatorLung hyperinflammationLung neutrophiliaNeutrophilic inflammationNeutrophil inflammationInflammation contributesLung damageNeutrophil recruitmentLung diseaseLung tissueReceptor 2Therapeutic targetNovel Approaches to Multidrug-Resistant Infections in Cystic Fibrosis
Murray T, Stanley G, Koff J. Novel Approaches to Multidrug-Resistant Infections in Cystic Fibrosis. Clinics In Chest Medicine 2022, 43: 667-676. PMID: 36344073, DOI: 10.1016/j.ccm.2022.06.008.Peer-Reviewed Original ResearchConceptsMultidrug-resistant organismsCystic fibrosisTherapeutic approachesNontuberculous mycobacteriaCystic fibrosis transmembrane conductance regulator (CFTR) dysfunctionSystemic adverse eventsRespiratory tract infectionsMethicillin-resistant Staphylococcus aureusAdditional clinical trialsNew treatment optionsPharmacokinetics/pharmacodynamicsInnovative therapeutic approachesMultidrug-resistant infectionsMDRO infectionAdverse eventsPulmonary infectionTract infectionsOptimal dosingTreatment optionsClinical trialsNew therapiesNegative organismsInfectionAntibiotic resistancePatientsUpdate in Advancing the Gastrointestinal Frontier in Cystic Fibrosis
Vélez C, Freedman S, Assis D. Update in Advancing the Gastrointestinal Frontier in Cystic Fibrosis. Clinics In Chest Medicine 2022, 43: 743-755. PMID: 36344078, DOI: 10.1016/j.ccm.2022.07.001.Peer-Reviewed Original ResearchNon-Modulator Therapies Developing a Therapy for Every Cystic Fibrosis Patient
Egan M. Non-Modulator Therapies Developing a Therapy for Every Cystic Fibrosis Patient. Clinics In Chest Medicine 2022, 43: 717-725. PMID: 36344076, DOI: 10.1016/j.ccm.2022.06.011.Peer-Reviewed Original ResearchMeSH KeywordsCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorGenetic TherapyHumansMutationConceptsModulator therapyCystic fibrosisCystic fibrosis transmembrane conductance regulator (CFTR) modulator therapiesCFTR modulator therapyTreatment of CFCystic fibrosis patientsGenetic-based therapiesMost patientsCF patientsFibrosis patientsTherapyPremature termination codon mutationsTherapeutic agentsPatientsDNA therapyRNA therapyTermination codon mutationsCodon mutationTransitions of Care in Cystic Fibrosis
DeFilippo E, Talwalkar J, Harris Z, Butcher J, Nasr S. Transitions of Care in Cystic Fibrosis. Clinics In Chest Medicine 2022, 43: 757-771. PMID: 36344079, DOI: 10.1016/j.ccm.2022.06.016.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsUpdate on Innate and Adaptive Immunity in Cystic Fibrosis
Bruscia E, Bonfield T. Update on Innate and Adaptive Immunity in Cystic Fibrosis. Clinics In Chest Medicine 2022, 43: 603-615. PMID: 36344069, DOI: 10.1016/j.ccm.2022.06.004.Peer-Reviewed Original ResearchMeSH KeywordsCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorHumansImmunity, InnateInflammationLungPneumoniaConceptsChronic infectionCFTR modulator therapyRobust inflammatory responseCystic fibrosis pathophysiologyImmune dysregulationPatient ageExcessive inflammationModulator therapyLung microenvironmentLung infectionImmune mechanismsInflammatory responseAdaptive immunityMucociliary transportCF life expectancyCF lungCystic fibrosisInfectionLife expectancyImmunityCritical roleCurrent understandingMorbidityInflammationFibrosisEmerging Concepts in Defective Macrophage Phagocytosis in Cystic Fibrosis
Jaganathan D, Bruscia EM, Kopp BT. Emerging Concepts in Defective Macrophage Phagocytosis in Cystic Fibrosis. International Journal Of Molecular Sciences 2022, 23: 7750. PMID: 35887098, PMCID: PMC9319215, DOI: 10.3390/ijms23147750.Peer-Reviewed Original ResearchMeSH KeywordsCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorHumansInflammationMacrophagesPhagocytosisConceptsPhagosome formationCystic fibrosis transmembrane conductance regulator (CFTR) geneTransmembrane conductance regulator geneInnate immunityTissue homeostasisRegulator geneMutant CFTRCF macrophagesCystic fibrosisPhagocytic mechanismsPathogenic microbesAdaptive immune systemDefective macrophage phagocytosisCFTRCurrent understandingTherapeutic developmentCentral roleMacrophage phagocytosisCFTR modulatorsPhagocytic cellsPhagocytosisNew therapeutic developmentsMacrophages contributesLung functionChronic inflammationColonic Fluid and Electrolyte Transport 2022: An Update
Negussie AB, Dell AC, Davis BA, Geibel JP. Colonic Fluid and Electrolyte Transport 2022: An Update. Cells 2022, 11: 1712. PMID: 35626748, PMCID: PMC9139964, DOI: 10.3390/cells11101712.Peer-Reviewed Original ResearchConceptsCystic fibrosis transmembrane regulatorCell death regulationSignal transduction pathwaysDeath regulationNa-K-Cl cotransporterEpithelial cell deathTransduction pathwaysIon homeostasisRegulatory relationshipsIon transportersTransepithelial ion transportEpithelial sodium channelTransmembrane regulatorCell deathColonic epithelial cellsEpithelial cellsTransportersSodium channelsNa-H exchangerRegulationIon transportCancer pathologyPH modulationIon movementOsmotic gradient
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