grant
Development of mini-USH2A gene therapy to treat Usher syndrome type 2A.
Organization SCHEPENS EYE RESEARCH INSTITUTELocation BOSTON, UNITED STATESPosted 1 May 2026Deadline 30 Apr 2028
NIHUS FederalResearch GrantFY2026AAV deliveredAAV deliveryAAV vectorAAV-based deliveryAAV-based vectorAAV-based viral deliveryAAV-mediated deliveryAddressAdeno-Associated VirusesAdeno-associated-virus-based deliveryAdvanced DevelopmentAnimal ModelAnimal Models and Related StudiesAnkleAuditoryAuditory Brainstem ResponsesBiochemicalBlindnessC-terminalCell LineCell modelCellLineCellular modelCochleaCochlear OrganCold-Insoluble GlobulinsComplexConeCorti CellCoupledDNA TherapyDNA mutationDataDeaf and blindDeafblindDefectDependoparvovirusDependovirusDevelopmentDiseaseDisorderEGFEGF geneFN1Fibronectin 1FibronectinsFutureGene DeliveryGene Transfer ClinicalGenesGenetic ChangeGenetic InterventionGenetic defectGenetic mutationGlycoprotein GP-2Hair CellsHearing LossHereditaryHumanHypoacusesHypoacusisImmunohistochemistryImmunohistochemistry Cell/TissueImmunohistochemistry Staining MethodIn VitroInheritedIntervening Protein SequenceInvestigationKO miceKnock-outKnock-out MiceKnockoutKnockout MiceLETS ProteinsLamininLarge External Transformation-Sensitive ProteinLeadLengthLinkMediatingMembraneMethodsMissionModern ManMutationNull MouseOpsinOpsonic GlycoproteinOpsonic alpha(2)SB GlycoproteinPathogenicityPatientsPb elementPhenotypePhotoreceptor CellPhotoreceptorsPhotosensitive CellPigmentary RetinopathyPreclinical dataProtein IntronsProteinsRPE65RPE65 proteinRegio tarsalisRepetitive ElementRepetitive RegionsRepetitive SequenceReportingResearchRetinaRetinal DefectRetinal anomaliesRetinitis PigmentosaRod-OpsinSensorineural DeafnessSensorineural Hearing LossSensory Hearing LossSightStrains Cell LinesSupplementationSystemTapetoretinal DegenerationTestingTherapeuticTimeTreatment EfficacyTubulinUSH2AUSH2A geneUsher 2AUsher Syndrome Type 2Usher Syndrome Type 2AUsher Syndrome Type IIUsher Type 2Usher Type IIVLGR1VLGR1 geneVariantVariationVestibular System ImpairmentVestibular defectVestibular dysfunctionVestibular problemsViral PackagingVirus PackagingsVisionVisual ReceptorWHRNWHRN geneWorkadeno associated virus groupadeno-associated viral vectoradeno-associated viral vector deliveryadeno-associated virus deliveryadeno-associated virus mediated deliveryadeno-associated virus vectoradenovirus mediated deliveryalpha 2-Surface Binding Glycoproteinautosomeclinical applicabilityclinical applicationclinical significanceclinically significantcultured cell linedeafnessdelivered with AAVdelivery with AAVdevelop therapydevelopmentaldysfunctional hearingear hair cellexon skippinggene repair therapygene therapygene-based therapygene-based treatmentgene-directed therapygene-targeted therapygene-targeted treatmentgenetic therapygenome mutationgenomic therapyhearing challengedhearing defecthearing deficienthearing deficithearing difficultyhearing dysfunctionhearing impairmenthearing restorationheavy metal Pbheavy metal leadin vivoinherited retinal degenerationinnovateinnovationinnovativeinsightinteinintervention developmentintervention efficacymembrane structureminiaturizeminiaturizedmodel of animalmouse modelmurine modelneuro-sensoryneurosensorynovelotoacoustic emissionpreclinical findingspreclinical informationpreventpreventingprotein biomarkersprotein markersreconstitutereconstitutionrestore hearingrod and cone dystrophyrod-cone dystrophyscreeningscreeningssensorineural hearing impairmenttargeted drug therapytargeted drug treatmentstargeted therapeutictargeted therapeutic agentstargeted therapytargeted treatmenttherapeutic efficacytherapy developmenttherapy efficacytreatment developmentvectorvestibular deficitvestibular impairmentvestibular system dysfunctionvision lossvisual functionvisual losswhirlin
Description preview
Project Summary
Usher syndrome type 2 (USH2) is the most common inherited condition for combined loss of hearing and vision
worldwide. Several pathogenic variants in the USH2A gene have been reported to cause USH2 and non-
syndromic retinitis pigmentosa (RP). The development of genetic therapies for USH2A has been mainly
hampered due to the large…
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