grant
Therapeutic potential of CC-90009 for cystic fibrosis
Organization UNIV OF NORTH CAROLINA CHAPEL HILLLocation CHAPEL HILL, UNITED STATESPosted 1 Sept 2024Deadline 31 Aug 2029
NIHUS FederalResearch GrantFY2025AML - Acute Myeloid LeukemiaAbbreviationsAcute Myeloblastic LeukemiaAcute Myelocytic LeukemiaAcute Myelogenous LeukemiaAirAnimal ModelAnimal Models and Related StudiesAnionsBiomedical ResearchCF miceCF mouse modelCFTRCFTR MouseCFTR ProteinCRISPR approachCRISPR based approachCRISPR methodCRISPR methodologyCRISPR techniqueCRISPR technologyCRISPR toolsCRISPR-CAS-9CRISPR-based methodCRISPR-based techniqueCRISPR-based technologyCRISPR-based toolCRISPR/CAS approachCRISPR/Cas methodCRISPR/Cas technologyCRISPR/Cas9CRISPR/Cas9 technologyCas nuclease technologyCell BodyCellsCellular biologyClimactericClustered Regularly Interspaced Short Palindromic Repeats approachClustered Regularly Interspaced Short Palindromic Repeats methodClustered Regularly Interspaced Short Palindromic Repeats methodologyClustered Regularly Interspaced Short Palindromic Repeats techniqueClustered Regularly Interspaced Short Palindromic Repeats technologyCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorCystic Fibrosis Transmembrane Conductance Regulator mouseDNA mutationDehydrationDiseaseDisorderDrugsE3 LigaseE3 Ubiquitin LigaseENaCEarly-Stage Clinical TrialsEpithelial CellsEpitheliumEquilibriumGenesGenetic ChangeGenetic DiseasesGenetic defectGenetic mutationHeightHereditaryHumanHydrationHydration statusHydrogen OxideIn VitroIn vivo analysisInbred CFTR MiceIndividualInflammationInheritedInnovative TherapyInvestmentsKnowledgeLiquid substanceLungLung Respiratory SystemLung infectionsMeasuresMedicalMedicationMentorsMessenger RNAMiceMice MammalsModern ManMolecularMorbidityMorbidity - disease rateMucociliary ClearanceMucociliary TransportMucous body substanceMucoviscidosisMucusMurineMusMutationObstructionOther GeneticsPathogenicityPathologicPathway interactionsPersonsPharmaceutical PreparationsPhase 1 Clinical TrialsPhase I Clinical TrialsPhase TransitionPhysiciansPositionPositioning AttributePre-Clinical ModelPreclinical ModelsProteinsPulmonary PathologyRegulator GenesResearch TechnicsResearch TechniquesResistanceRespiratory EpitheliumRibosomesS PeriodS phaseScientistSedalisServicesStop CodonStructure of respiratory epitheliumSurfaceSymptomsSynthesis PeriodSynthesis PhaseSystemTermination CodonTerminator CodonTestingThalidomideTherapeuticTracheal EpitheliumTranscriptional Regulatory ElementsTransgenic OrganismsTranslation Stop SignalTranslationsUbiquitilationUbiquitin Protein LigaseUbiquitin-Protein Ligase ComplexesUbiquitin-Protein Ligase E3UbiquitinationUbiquitinoylationVariantVariationWaterWorkacute granulocytic leukemiaacute myeloid leukemiaairway epitheliumbalancebalance functionbiophysical characteristicsbiophysical characterizationbiophysical measurementbiophysical parametersbiophysical propertiesbody water dehydrationbronchial epitheliumcareercell biologycystic fibrosis mousecystic fibrosis mouse modelcystic fibrosis transmembrane regulatordrug/agentepithelial Na+ channelepithelial sodium channelfluidgenetic conditiongenetic disordergenetic trans acting elementgenome mutationimprovedimproved outcomein vivo evaluationin vivo testinginnovateinnovationinnovativeknock-downknockdownlife changeliquidlung pathologymRNAmodel of animalmortalitymouse modelmuco obstructive lung diseasesmucoobstructive lung diseasesmucousmucus-associated lung diseasesmurine modelmutantnew drug treatmentsnew drugsnew pharmacological therapeuticnew therapeuticsnew therapynext generation therapeuticsnovelnovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel therapeuticsnovel therapyoverexpressoverexpressionpathwayphase I protocolpre-clinicalpreclinicalprematureprematuritypulmonary infectionsregulatory generesistantrespiratoryrespiratory tract epitheliumribosome recycling factorribosome releasing factorsmall moleculetermination factortherapeutic targettrans acting elementtransgenictranslationtranslational cliniciantranslational opportunitiestranslational physiciantranslational potentialtranslational therapeuticstranslational therapyubiquinationubiquitin conjugationubiquitin-protein ligase
Description preview
PROJECT SUMMARY/ABSTRACT
Cystic fibrosis (CF) is an inherited, multisystem disease caused by mutations in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene that encodes the CFTR epithelial anion channel. CFTR
works in balance with the epithelial sodium channel (ENaC) to maintain hydration of the airway surface.
Perturbations of…
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