grant

Auditory circuit organization following gene therapy for congenital deafness

Organization JOHNS HOPKINS UNIVERSITYLocation BALTIMORE, UNITED STATESPosted 1 Aug 2025Deadline 31 Jul 2028
NIHUS FederalResearch GrantFY20250-11 years old2-photon21+ years oldAAV vectorAAV-based vectorAcoustic NerveAddressAdultAdult HumanAffectAuditoryAuditory CortexAuditory LocalizationAuditory areaAuditory systemBrainBrain Nervous SystemCNS plasticityCalciumCell BodyCell Communication and SignalingCell SignalingCellsChildChild YouthChildren (0-21)ClinicalClinical TrialsCochleaCochlear ImplantsCochlear OrganCochlear ProsthesisComplexCorti CellCranial Nerve EightCranial Nerve VIIIDNA TherapyDarknessDevelopmentEarEarly InterventionEighth Cranial NerveEncephalonEsthesiaGene Transfer ClinicalGene variantGenetic DiseasesGenetic InterventionHair CellsHearingImageImaging ProceduresImaging TechnicsImaging TechniquesIndividualInferior ColliculusInner Hair CellsInner ear hair cellsInternal EarInterventionIntracellular Communication and SignalingKO miceKnock-out MiceKnockout MiceLabyrinthLinguisticLinguisticsMediatingMethodsMiceMice MammalsModalityMonitorMurineMusNerve CellsNerve FibersNerve Impulse TransmissionNerve TransmissionNerve UnitNeural CellNeural PathwaysNeurocyteNeuronal PlasticityNeuronal TransmissionNeuronsNull MouseOTOF geneOTOF proteinOlder PopulationOrganizational ChangePatient outcomePatient-Centered OutcomesPatient-Focused OutcomesPatientsPatternPeripheralPosterior Quadrigeminal BodyProcessPropertyRecoveryRegimenRehabilitation OutcomeResearchRoleSensationSensorySensory DeprivationSensory HairShapesSignal TransductionSignal Transduction SystemsSignalingSound LocalizationSpeech PerceptionStructureSynapsesSynapticSynaptic VesiclesTherapeutic InterventionTimeTransmissionVIIIth Cranial NerveVariantVariationVesicleVestibulocochlear Nerveadeno-associated viral vectoradeno-associated virus vectoradulthoodallelic variantauditory nerveauditory pathwayauditory rehabilitationaxon signalingaxon-glial signalingaxonal signalingbiological signal transductioncentral nervous system plasticitycongenital deafnesscongenitally deafcritical perioddeafdeafeneddeafnessdeprivationdevelopmentalear hair cellfunctional restorationgene repair therapygene therapygene-based therapygenetic conditiongenetic disordergenetic therapygenetic variantgenomic therapygenomic variantglia signalingglial signalinghearing restorationimagingimprovedimproved outcomeinner earinsightintervention therapykidsnerve signalingneuralneural plasticityneural signalingneuronalneuronal signalingneuroplasticneuroplasticityneurotransmissionneurotransmitter releasenew approachesnovel approachesnovel strategiesnovel strategyolder groupsolder individualsolder personotoferlinpatient oriented outcomesprofound hearing lossprotein functionrehabilitative outcomeresponserestorationrestore functionrestore functionalityrestore hearingrestore lost functionsensorsocial rolesoundsynapsetransmission processtwo-photonvisual deprivationyoungster
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PROJECT SUMMARY
This project seeks to understand the critical role of otoferlin in auditory circuit development and how gene

therapy can be optimized to restore hearing in patients with congenital deafness caused by otoferlin variants.

Otoferlin is a key calcium sensor that facilitates synaptic vesicle fusion and neurotransmitter release at inner…

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Auditory circuit organization following gene therapy for congenital deafness — JOHNS HOPKINS UNIVERSITY | UNITED STATES | Dev Procure