grant
The Impact of SLC26A9 on Response to CFTR Correction
Organization CINCINNATI CHILDRENS HOSP MED CTRLocation CINCINNATI, UNITED STATESPosted 15 Aug 2025Deadline 31 May 2030
NIHUS FederalResearch GrantFY2025AAV vectorAAV-based vectorAddressAirway healthAutoregulationBicarbonatesBiologicalBronchiectasisCF lung diseaseCF patientsCFTRCFTR ProteinCOPDCaringCell LineCell modelCellLineCellular modelChildhoodChloridesChronic Obstruction Pulmonary DiseaseChronic Obstructive Lung DiseaseChronic Obstructive Pulmonary DiseaseClinical DataCohort StudiesConcurrent StudiesCoupledCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDNA TherapyDataData AnalysesData AnalysisDecrease disparityDelta F508 mutationDependenceDiseaseDisorderDrug ModelingsDrug TherapyDrugsEndocrine DiseasesEndocrine Diseases and ManifestationsEndocrine System DiseasesEpithelial PhysiologyF508 deletionF508 mutationF508-delF508delFEV1FEV1%VCFailureFluid BalanceFluid HomeostasisForced Expiratory Volume 1 TestForced Expiratory Volume in 1 SecondFrequenciesFutureGastrointestinal DiseasesGene DeliveryGene FrequencyGene Transfer ClinicalGenesGenetic InterventionGenotypeGoalsHCO3HeterogeneityHomeostasisHydrationHydration statusHydrogen CarbonatesImpairmentIn VitroIndividualIonsLinkLiquid substanceLower disparityMedicationModificationMolecularMucociliary ClearanceMucociliary TransportMucoviscidosisOutcomePFT/FEV1Pancreatic DiseasesPancreatic DisorderPatientsPharmaceutical PreparationsPharmacological TreatmentPharmacotherapyPhysiologicPhysiologicalPhysiological HomeostasisPhysiologyPopulationPositionPositioning AttributePrecision careProbabilistic ModelsProbability ModelsPublic HealthPulmonary Cystic FibrosisPulmonary Function Test/Forced Expiratory Volume 1QOLQuality of lifeRegulationRespiratory EpitheliumRiskRisk-associated variantRoleSeveritiesSeverity of illnessStatistical ModelsStrains Cell LinesStructure of respiratory epitheliumTherapeuticTherapeutic AgentsTranslational ResearchTranslational ScienceVariantVariationWorkadeno-associated viral vectoradeno-associated virus vectorairway epitheliumairway injuryairway surface liquidallelic frequencybase editingbiologicburden of diseaseburden of illnesschronic obstructive pulmonary disorderco-morbidco-morbiditycohortcomorbiditycultured cell linecystic fibrosis lungcystic fibrosis lung diseasecystic fibrosis patientscystic fibrosis transmembrane regulatordata interpretationdecline in functiondecline in functional statusdisease burdendisease severitydisparity reductiondrug interventiondrug treatmentdrug/agentendocrine disorderexperiencefluidfunctional declinefunctional status declinegastrointestinal disordergene repair therapygene replacementgene replacement therapygene therapygene-based therapygenetic therapygenomic therapyhuman subjectimpaired airwayimprovedindividual patientindividual responseindividualized careindividualized patient careindividualized responseindividuals with CFindividuals with cystic fibrosisinjured airwayliquidlung functionlung function declinemitigate disparitymuco obstructive lung diseasesmucoobstructive lung diseasesmucus-associated lung diseasesnovelpancreas disorderpatients with CFpatients with cystic fibrosispediatricpersonalization of treatmentpersonalized carepersonalized medicinepersonalized patient carepersonalized therapypersonalized treatmentpharmaceutical interventionpharmacological interventionpharmacological therapypharmacology interventionpharmacology treatmentpharmacotherapeuticsprotective alleleprotective variantpulmonary functionpulmonary function declinerecruitreduce disparityreduction in disparityrespiratoryrespiratory healthrespiratory injuryrespiratory tract epitheliumrespiratory tract injuryresponders and non-respondersresponders from non-respondersresponders or non-respondersresponders versus non-respondersresponders vs non-respondersresponders/nonrespondersresponseresponse to therapyresponse to treatmentrestorationrisk allelerisk generisk genotyperisk locirisk locusrisk stratificationrisk variantsocial rolesolutestatistical linear mixed modelsstatistical linear modelsstratify risktherapeutic responsetherapy responsetraffickingtranslation researchtranslational investigationtreatment responsetreatment responsivenessΔF508
Description preview
PROJECT ABSTRACT / SUMMARY
Although CFTR modulator therapies have improved respiratory outcomes for the Cystic Fibrosis (CF) population,
the benefit to individual patients can vary widely. In “real world” studies of modulator therapies, patients continue
to experience lung function decline, and as many as 1 in 5 receive no therapeutic benefit in…
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