grant
AAV-mediated editing to treat human autosomal dominant hearing loss DFNA41 and DFNA2
Organization MASSACHUSETTS EYE AND EAR INFIRMARYLocation BOSTON, UNITED STATESPosted 20 Sept 2024Deadline 31 Aug 2028
NIHUS FederalResearch GrantFY20250-11 years old0-4 weeks old21+ years oldAAV deliveredAAV deliveryAAV-based deliveryAAV-based viral deliveryAAV-mediated deliveryAdeno-Associated VirusesAdeno-associated-virus-based deliveryAdultAdult HumanAffectAnimal ModelAnimal Models and Related StudiesAnimalsAreaBehaviorBilateral DeafnessBiodistributionBiological Response Modifier TherapyBiological TherapyBody TissuesC3H MouseCMV promoterCRISPR based therapeuticsCRISPR based treatmentCRISPR therapeuticsCRISPR treatmentCRISPR-Cas based therapeuticsCRISPR-based disease therapeuticsCRISPR-based therapyCRISPR/Cas therapeuticsCRISPR/Cas9 therapeuticsCRISPR/Cas9 therapyCRISPR/Cas9 treatmentCRISPR/Cas9-based therapyCas9 based therapeuticsCell BodyCell LineCell SurvivalCell ViabilityCellLineCellsChildChild YouthChildren (0-21)ClinicClinical ResearchClinical StudyClinical TrialsClustered Regularly Interspaced Short Palindromic Repeats based therapeuticsClustered Regularly Interspaced Short Palindromic Repeats therapeuticsCochlear ImplantsCochlear ProsthesisCommunitiesComplete DeafnessComplete Hearing LossComplexCorti CellCrab-Eating MacaqueCrab-Eating MonkeyCynomolgus MonkeyCynomolgus macaqueDNA Damage RepairDNA RepairDNA TherapyDNA mutationDataDependoparvovirusDependovirusDevelopmentDiseaseDisorderDominant Genetic ConditionsDominant traitDoseDrug TherapyEarFDA approvedFamily suidaeFeedbackFoundationsGene Transfer ClinicalGenesGenetic ChangeGenetic DiseasesGenetic DominantGenetic InterventionGenetic defectGenetic mutationGenomeGoalsGuide RNAHair CellsHearingHearing LossHumanHuman Cell LineHuman GeneticsHypoacusesHypoacusisInbred C3H MiceInternal EarKnowledgeLabyrinthM fascicularisM. fascicularisMacaca cynomolgusMacaca fascicularisMacaca irusMediatingMiceMice MammalsMinimal Access Surgical ProceduresMinimally Invasive Surgical ProceduresModern ManMurineMusMutant Strains MiceMutationNatural HistoryNewborn InfantNewbornsNon-Polyadenylated RNAOTOF geneOTOF proteinPatientsPharmacological TreatmentPharmacotherapyPigsPopulationProcessQuality ControlRNARNA Gene ProductsRibonucleic AcidRouteSafetySensory HairSpeedStrains Cell LinesSuidaeSwineTechnologyTestingTherapeuticTissuesTotal DeafnessToxic effectToxicitiesTranslationsTreatment EfficacyUnscheduled DNA SynthesisValidationVestibular epitheliaVestibular inner earVirus IntegrationWorkadeno associated virus groupadeno-associated viral vector deliveryadeno-associated virus deliveryadeno-associated virus mediated deliveryadenovirus mediated deliveryadulthoodautosomebiological therapeuticbiological treatmentbiologically based therapeuticsbiotherapeuticsbiotherapycandidate identificationcell immortalizationcell transductioncellular transductioncongenital hearing impairmentcongenital hearing losscultured cell linedeafdeafeneddeafnessdelivered with AAVdelivery vectordelivery vehicledelivery with AAVdesigndesigningdevelopmentaldominant genetic mutationdominant mutationdrug interventiondrug treatmentdysfunctional hearingear hair cellefficacy studyexperiencegRNAgene repair therapygene replacement therapygene therapygene therapy clinical trialgene-based therapygenetic conditiongenetic disordergenetic hearing impairmentgenetic hearing lossgenetic therapygenome analysisgenome editinggenome mutationgenomic editinggenomic therapyhearing challengedhearing defecthearing deficienthearing deficithearing difficultyhearing dysfunctionhearing impairmenthearing restorationhereditary hearing impairmenthereditary hearing losshuman modelin vivoinherited hearing impairmentinherited hearing lossinner earintervention efficacykidslong-tailed macaquemanufacturemeetingmeetingsmodel of animalmodel of humanmouse modelmouse mutantmurine modelnew drug treatmentsnew drugsnew pharmacological therapeuticnew therapeuticsnew therapynewborn childnewborn childrennext generation therapeuticsnon-human primatenonhuman primatenovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel therapeuticsnovel therapyoff-target siteotoferlinpharmaceutical interventionpharmacological interventionpharmacological therapypharmacology interventionpharmacology treatmentpharmacotherapeuticspig modelpiglet modelporcineporcine modelpre-clinical studypreclinical studyprofound hearing lossprogressive hearing lossrestore hearingsafety studysuidswine modelsystemic toxicitytherapeutic efficacytherapy efficacytransduced cellstransduction efficiencytranslationvalidationsvectorviral genome integrationviral integrationvirus genome integrationyoungster
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Project Summary/Abstract
1 in 500 newborns suffers from genetic hearing loss (HL), and there are no FDA-approved drugs or biological treatments
for any type of HL. Genome editing that disrupts or repairs DNA mutations or modulates RNA levels is being developed
into new therapies for many diseases. Despite the unprecedented progress, the…
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