grant
Models for Therapy of Hereditary Retinal Degeneration
Organization UNIVERSITY OF PENNSYLVANIALocation PHILADELPHIA, UNITED STATESPosted 1 Dec 1992Deadline 31 Dec 2029
NIHUS FederalResearch GrantFY2025AAV deliveredAAV deliveryAAV-based deliveryAAV-based viral deliveryAAV-mediated deliveryAccelerationAddressAdeno-associated-virus-based deliveryAffectAgeAnimal DiseasesAnimal ModelAnimal Models and Related StudiesAutosomal Recessive Medullary Cystic DiseaseBest DiseaseBiologic ModelsBiologicalBiological ModelsBlindnessBreedingCanine SpeciesCanis familiarisCell BodyCell TherapyCellsCellular biologyCiliaClinicClinicalClinical TrialsCollaborationsCooperative AgreementsDNA TherapyDNA mutationDevelopmentDiseaseDisorderDogsDogs MammalsEnsureFosteringFundingFutureGene Transfer ClinicalGenesGeneticGenetic ChangeGenetic InterventionGenetic defectGenetic mutationGenotypeGoalsGrantHealthHereditaryHumanIndustryInheritedInjectionsInvestigationInvestigatorsLeber congenital amaurosisLeber's amaurosisLeber's congenital amaurosisMacromolecular StructureMarketingMethodsModel SystemModelingModern ManMolecularMolecular StructureMolecular TargetMonitorMutationNPHPNational Institutes of HealthNephronophthisisOphthalmologyOutcomeOutcome MeasureOuter pigmented layer of retinaPathogenesisPathogenicityPatientsPennsylvaniaPersonsPhenotypePhotoreceptor CellPhotoreceptorsPhotosensitive CellPigment cell layer of retinaPigmentary RetinopathyPigmented layer of retinaPreclinical dataPredictive ValueProductivityRPE65RPE65 proteinRPGRRPGR geneResearchResearch PersonnelResearch ProposalsResearch ResourcesResearchersResourcesRetinaRetinal DegenerationRetinal DiseasesRetinal DisorderRetinal Pigment EpitheliumRetinal pigment epithelial cellsRetinitis PigmentosaRetinitis Pigmentosa GTPase RegulatorRodRouteSafetySeriesSiteSolidStructureStructure of retinal pigment epitheliumTapetoretinal DegenerationTestingTherapeutic InterventionTherapeutic StudiesTherapy ResearchU-Series Cooperative AgreementsUnited States National Institutes of HealthUniversitiesVisualVisual ReceptorVitelliform MDVitelliform macular dystrophyadeno-associated viral vector deliveryadeno-associated virus deliveryadeno-associated virus mediated deliveryadenovirus mediated deliveryagesamaurosis congenita of Leberbest macular dystrophybiologiccaninecanine animal modelcanine modelcell based interventioncell biologycell mediated interventioncell mediated therapiescell replacement therapycell replacement treatmentcell typecell-based therapeuticcell-based therapycellular therapeuticcellular therapyciliopathyclinical relevanceclinically relevantcone-rod dystrophycongenital amaurosis of retinal origincost effectivedegenerative retina diseasesdelivered with AAVdelivery with AAVdesigndesigningdetermine efficacydevelop therapydevelopmentaldisease modeldisease preventiondisorder modeldisorder preventiondog modeldomestic dogeffective therapyeffective treatmenteffectiveness testingefficacy analysisefficacy assessmentefficacy determinationefficacy evaluationefficacy examinationend stage diseaseevaluate efficacyexamine efficacyexperiencegene repair therapygene replacement therapygene therapygene-based therapygenetic therapygenome mutationgenomic therapyhuman diseaseimprovedintervention developmentintervention therapymanmeasurable outcomemodel of animalmutantnew approachesnew drug treatmentsnew drugsnew pharmacological therapeuticnew therapeutic approachnew therapeutic interventionnew therapeutic strategiesnew therapeuticsnew therapynew therapy approachesnew treatment approachnew treatment strategynext generation therapeuticsnovelnovel approachesnovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel strategiesnovel strategynovel therapeutic approachnovel therapeutic interventionnovel therapeutic strategiesnovel therapeuticsnovel therapynovel therapy approachoptogeneticsoutcome measurementphotoreceptor degenerationpre-clinicalpre-clinical evaluationpreclinicalpreclinical evaluationpreclinical findingspreclinical informationpredict responsivenesspredicting responsepreventpreventingprogenitor cell based therapyprogenitor cell therapyprogenitor cell treatmentprogenitor therapyprogenitor treatmentprogramsreproductiveresearch facilityresearch studyresponseresponse to therapyresponse to treatmentrestore sightrestore visionretina degenerationretina diseaseretina disorderretinal degenerativeretinal degenerative diseasesretinopathyrod and cone dystrophyrod-cone dystrophysight restorationsmall moleculesoundstem and progenitor cell therapystem cell based therapystem cell mediated therapystem cell therapeuticsstem cell therapystem cell treatmentstem cell-based therapeuticstem cell-based treatmentsuccesstherapeutic outcometherapeutic responsetherapeutic targettherapy developmenttherapy outcometherapy responsetranslational studytreatment developmenttreatment responsetreatment responsivenessvision lossvision restorationvisual loss
Description preview
Grant EY006855 Summary
Inherited retinal diseases (IRDs) such as retinitis pigmentosa, Leber congenital amaurosis, cone-rod
dystrophy, and Best macular dystrophy are devastating blinding diseases in people. While mutations in nearly
270 genes have been associated with different forms of IRDs to date, characterization of disease mechanisms
and…
🔒
Start 7-day free trial — $29.99/mo →Full details available on the Agency plan
Unlock the complete grant description, eligibility criteria, contract value, evaluation details and apply link — plus alerts, pipeline tracking, and CSV export.
Agency Plan
7-day free trialUnlock procurement & grants
Upgrade to access active tenders from World Bank, UNDP, ADB and more — with email alerts and pipeline tracking.
$29.99 / month
- 🔔Email alerts for new matching tenders
- 🗂️Track tenders in your pipeline
- 💰Filter by contract value
- 📥Export results to CSV
- 📌Save searches with one click