grant
Engineering AAV for safe and efficient gene delivery to the human retina
Organization UNIVERSITY OF FLORIDALocation GAINESVILLE, UNITED STATESPosted 1 Jun 2014Deadline 30 Jun 2029
NIHUS FederalResearch GrantFY2025AcademiaAcuteAddressAdeno-Associated VirusesAdrenal Cortex HormonesAge related macular degenerationAge-Related MaculopathyAnatomic SitesAnatomic structuresAnatomyAnimalsAntigen-Presenting CellsAreaAssociated VirusesAttentionBasic ResearchBasic ScienceBenchmarkingBest Practice AnalysisBindingBiodistributionBiologicalBlebBlisterBody TissuesBullaBullous LesionBypassCapsidCell BodyCell NucleusCellsCharacteristicsClinicClinicalClinical TrialsCommunitiesCorticoidsCorticosteroidsDNA TherapyDataDependenceDependoparvovirusDependovirusDiabetic RetinopathyDirected Molecular EvolutionEngineeringEnsureEyeEyeballFoveaFundingGene DeliveryGene Transfer ClinicalGenetic InterventionGlaucomaGlycansGoalsHSPGHeparan Sulfate ProteoglycanHereditaryHost FactorHost Factor ProteinHumanImmuneImmune systemImmunesIn SituIndustryInflammationInheritedInner Limiting MembraneIntegration Host FactorsJuvenile RetinoschisisKnowledgeLeber congenital amaurosisLeber's amaurosisLeber's congenital amaurosisLibrariesMALD-MSMALDIMALDI-MSMacacaMacaqueMass Photometry/Spectrum AnalysisMass SpectrometryMass SpectroscopyMass SpectrumMass Spectrum AnalysesMass Spectrum AnalysisMedicalMethodsMiceMice MammalsModelingModern ManMolecular InteractionMuller gliaMuller's cellMurineMusMüller cellMüller gliaN-Acetylneuraminic AcidsNatureNucleusOperative ProceduresOperative Surgical ProceduresPatientsPeripheralPhase 1/2 Clinical TrialPhase 1/2 trialPhase I/II Clinical TrialPhase I/II TrialPhotoreceptor CellPhotoreceptorsPhotosensitive CellPolysaccharidesPre-clinical Drug Testing/DevelopmentPreclinical Drug DevelopmentPreclinical Drug Testing/DevelopmentPrimatesPrimates MammalsProteoheparan SulfateRPE65RPE65 proteinReceptor ProteinResource SharingRetinaRetinal DiseasesRetinal DisorderRetinal Ganglion CellsRetinal gene therapyRetinoschisis, Juvenile, X Chromosome-LinkedRetinoschisis, Juvenile, X-LinkedRodentRodentiaRodents MammalsRoleRouteSafetySatellite VirusesSialic AcidsSightSpectrometry, Mass, Matrix-Assisted Laser Desorption-IonizationSpectroscopy, Mass, Matrix-Assisted Laser Desorption-IonizationSurgicalSurgical InterventionsSurgical ProcedureT cell responseTimeTissuesTranslatingTranslational ResearchTranslational ScienceTreatment EfficacyVariantVariationVesicationViralViral ReceptorVirus ReceptorsVisionVisual ReceptorVisualizationWorkX-Linked Retinoschisisaccessory celladeno associated virus groupage dependent macular degenerationage induced macular degenerationage related macular diseaseage related macular dystrophyamaurosis congenita of Leberbenchmarkbiologiccongenital amaurosis of retinal origindesigndesigningdevelop therapydirected evolutionfovea centralisgene repair therapygene therapygene-based therapygenetic therapygenomic therapygeographic atrophyglaucomatoushydrophilicityimmune system functionimprovedintervention developmentintervention efficacyintravitreal injectionmaculamacularmatrix assisted laser desorption ionizationmolecular imagingmolecule imagingneurotropicneutralizing antibodynon-human primatenonhuman primatenovelpre-clinical drug developmentrational designreceptorretina diseaseretina disorderretinal ganglionretinopathyscreeningscreeningssenile macular diseasesocial rolesubretinal injectionsurgerytherapeutic efficacytherapy developmenttherapy efficacytool developmenttraffickingtransduction efficiencytransgene expressiontranslation researchtranslational investigationtreatment developmentvectorvisual function
Description preview
ABSTRACT
This is a competitive renewal of R01EY024280, “Engineering AAV for safe and efficient gene delivery to the
human retina”. FDA approval of an Adeno associated virus (AAV)- based gene therapy for RPE65-Leber
congenital amaurosis (LCA2) solidified gene therapy’s place in current medical practice. However, in the 6 years
since, there have…
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