grant
CFTR gene correction via targeted exon replacement
Organization UNIVERSITY OF TEXAS AT AUSTINLocation AUSTIN, UNITED STATESPosted 1 Jan 2026Deadline 31 Dec 2028
NIHUS FederalResearch GrantFY2025AffectAmino AcidsAnionsBiochemicalCF patientsCFTRCFTR ProteinCell BodyCell LineCellLineCellsChromosomal RearrangementChromosomal dislocationChromosomal translocationChronicClassificationCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDNADNA Repair PathwayDNA TherapyDNA editorDNA mutationDataDeoxyribonucleic AcidDiseaseDisorderEC 2.7.7.49ElectroporationEpithelial CellsEpitheliumExhibitsExonsFDA approvedFaceFamilyGene Transfer ClinicalGenesGenetic ChangeGenetic DiseasesGenetic InterventionGenetic TranslocationGenetic defectGenetic mutationGenomeGuide RNAHealth Care CostsHealth CostsHumanIndividualLengthLettersLifeMeasuresMediatingMembraneMendelian diseaseMendelian disorderMendelian genetic disorderMessenger RNAModalityModern ManModificationMucoviscidosisMutationNon-Polyadenylated RNAOrangesOutcomePathogenicityPatientsPersonsRNARNA Gene ProductsRNA SplicingRNA TranscriptaseRNA-Dependent DNA PolymeraseRNA-Directed DNA PolymeraseReverse TranscriptaseRevertaseRibonucleic AcidRiskSingle-Stranded DNASiteSplicingStrains Cell LinesSystematicsTestingTherapeuticTherapeutic Gene EditingTimeWorkadverse consequenceadverse outcomeaminoacidbase editorbronchial epitheliumburden of diseaseburden of illnesscell immortalizationchromosome dislocationchromosome translocationclinical relevanceclinically relevantcultured cell linecystic fibrosis patientscystic fibrosis transmembrane regulatordisease burdenelectroporative deliveryfacesfacialgRNAgene correctedgene correctiongene editing methodgene editing methodologygene editing strategygene editing techniquesgene editorgene electrotransfergene repair therapygene therapygene-based therapygene-editing approachgene-editing therapygenetic conditiongenetic disordergenetic therapygenome editing based therapygenome editing therapygenome editing treatmentgenome editing-based therapeuticsgenome editorgenome mutationgenomic correctiongenomic therapyimprovedindividuals with CFindividuals with cystic fibrosismRNAmRNA Decaymembrane structuremonogenic diseasemonogenic disordernucleasepatients with CFpatients with cystic fibrosisrepairrepairedrestorationsingle-gene diseasesingle-gene disordersmall moleculessDNAstoichiometrytherapeutic editingtherapeutic genome editing
Description preview
PROJECT SUMMARY
Cystic fibrosis (CF) is a life-threatening genetic disorder that affects >120,000 individuals worldwide. Mutations
in the CFTR gene cause CF, with 10% of people with CF (pwCF) having two copies of mutations that are unre-
sponsive to FDA-approved CFTR modulators. Exon 12 contains the most common modulator-ineligible mutations,…
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