grant
Cystic Fibrosis Gene Therapy with adeno-associated viral vectors
Organization JOHNS HOPKINS UNIVERSITYLocation BALTIMORE, UNITED STATESPosted 1 Jul 2024Deadline 30 Apr 2029
NIHUS FederalResearch GrantFY2025AAV vectorAAV-based vectorAddressAffectAnimal ModelAnimal Models and Related StudiesBasal CellBlood CirculationBloodstreamBody TissuesCF patientsCFTRCFTR ProteinCapsidCell BodyCellsCharacteristicsChickensClinicalClinical TrialsCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDNA TherapyDNA mutationDefectDelta F508 mutationDevelopmentDirected Molecular EvolutionDiseaseDisorderDoseEarly-Stage Clinical TrialsF508 deletionF508 mutationF508-delF508delFerretsGallus domesticusGallus gallusGallus gallus domesticusGene ExpressionGene Transfer ClinicalGene therapy trialGenesGenetic ChangeGenetic InterventionGenetic defectGenetic mutationGoalsHealthHumanIndividualInvestigatorsLungLung DiseasesLung Respiratory SystemM mulattaM. mulattaMacaca mulattaMacaca rhesusMeasuresModelingModern ManMonkeysMorbidityMorbidity - disease rateMucoviscidosisMutationNHP modelsNatureOrganPathologyPatientsPharmacology and ToxicologyPhase 1 Clinical TrialsPhase I Clinical TrialsPhenotypeProcessProteinsPulmonary DiseasesPulmonary DisorderRecombinant adeno-associated virusRecombinant adeno-associated virus (rAAV)ReproducibilityResearch PersonnelResearchersRhesus MacaqueRhesus MonkeySafetySerotypingSurfaceTherapeutic AgentsTherapeutic EffectTimeTissuesTracheaTrachea ProperTransduction GeneTropismVector Mediated Transfer GenesViralViral VectorVirusWorkadeno-associated viral vectoradeno-associated virus vectorautosomebeta Actincell typecystic fibrosis patientscystic fibrosis transmembrane regulatordesigndesigningdevelopmentaldirected evolutiondisease of the lungdisease phenotypedisorder of the lungeffective therapyeffective treatmentfunctional restorationgene repair therapygene therapeuticsgene therapygene transfer trialgene-based therapeuticgene-based therapeuticsgene-based therapygenes therapeuticgenes therapeuticsgenetic therapygenome mutationgenomic therapyindividuals with CFindividuals with cystic fibrosislung disordermodel of animalmortalitymutantnew approachesnonhuman primate modelsnovelnovel approachesnovel strategiesnovel strategypatients with CFpatients with cystic fibrosisphase I protocolpre-clinical trialpreclinical trialpromoterpromotorrAAVrecombinant AAVrestore functionrestore functionalityrestore lost functiontherapeutic genetransgene expressionvectorwindpipeΔF508β-Actin
Description preview
Abstract: We and others have shown that AAV vectors have great potential as gene therapeutic agents (3-5),
in particular for CF. Studies originating from our group led to the first use of rAAV in humans (7). Many pre-
clinical and clinical trials have shown that AAV vectors can be used safely (5,6,9). The major challenge, however,
is that they…
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