grant

Gene therapy targeting CFH and lipoprotein dynamics in AMD

Organization DUKE UNIVERSITYLocation DURHAM, UNITED STATESPosted 1 Sept 2025Deadline 30 Jun 2028
NIHUS FederalResearch GrantFY2025APOEAffectAgeAge related macular degenerationAge-Related MaculopathyAgingAlternative Complement PathwayAmericanAmino AcidsAnimal ModelAnimal Models and Related StudiesAnimalsApo A-1Apo A-IApo A1Apo AIApo-BApo-EApoA-1ApoA-IApoBApoE proteinApolipoprotein A-1Apolipoprotein A-IApolipoprotein A1Apolipoprotein AIApolipoprotein EApolipoproteins BAreaBindingBiologic ModelsBiological ModelsBlindnessBloodBlood PlasmaBlood Reticuloendothelial SystemBody TissuesBruch MembraneBruch's basal membrane structureBruch's membraneBruchs membraneCell BodyCellsCessation of lifeCholesterolChoroidComplementComplement ActivationComplement Factor HComplement ProteinsComplexDNA TherapyDataDeathDepositDepositionDeveloped CountriesDevelopmentDietDiseaseDisorderDruseDrusenDysfunctionEconomic BurdenElderlyEngineeringEpidemiologic ResearchEpidemiologic StudiesEpidemiological StudiesEpidemiological dataEpidemiology ResearchEpidemiology dataEpithelial CellsEquilibriumEyeEye diseasesEyeballFactor HFatsFatty acid glycerol estersFunctional disorderGene Transfer ClinicalGenesGenetic InterventionGenetic PolymorphismGenetic RiskGenetic studyGlycosaminoglycansGoalsHDLHDL LipoproteinsHeavy LipoproteinsHigh Density LipoproteinsHistidineHumanIn VitroIncidenceIndustrialized CountriesIndustrialized NationsInflammatoryIntermediary MetabolismLamina Basalis ChoroideaeLengthLesionLinkLipidsLipoprotein BindingLipoproteinsMediatingMetabolic ProcessesMetabolismMiceMice MammalsModalityModel SystemModelingModern ManMolecular InteractionMucopolysaccharidesMurineMusNational Eye InstituteOcular PathologyOutcome StudyOuter pigmented layer of retinaPathogenesisPathogenicityPathologyPathway interactionsPatientsPhenotypePhysiopathologyPigment EpitheliumPigment cell layer of retinaPigmented layer of retinaPlasmaPlasma SerumPlayPositionPositioning AttributePreclinical dataProperdin PathwayPropertyProteinsProteomeRecombinant adeno-associated virusRecombinant adeno-associated virus (rAAV)ResearchResearch PriorityReticuloendothelial System, Serum, PlasmaRetinal Pigment EpitheliumRetinal pigment epithelial cellsRiskRisk FactorsRisk-associated variantRoleSerotypingStructure of retinal pigment epitheliumTestingTissuesTransgenesTyrosineVariantVariationadvanced ageage associatedage associated alterationsage associated changesage correlatedage correlated alterationsage correlated changesage dependentage dependent alterationsage dependent changesage dependent macular degenerationage induced alterationsage induced changesage induced macular degenerationage linkedage relatedage related alterationsage related changesage related macular diseaseage related macular dystrophyage specificage specific alterationsage specific changesagedaged miceaged mouseagesaging associated alterationsaging associated changesaging correlated alterationsaging correlated changesaging dependent alterationsaging dependent changesaging induced alterationsaging induced changesaging related alterationsaging related changesaging specific alterationsaging specific changesalpha-Lipoproteinsalterations with ageaminoacidapo B-48apoB-48apoB48apolipoprotein B-48apolipoprotein B48apoprotein B-48balancebalance functionchanges with agecomplement pathway regulationcomplement systemcomplementationdesigndesigningdeveloped countrydeveloped nationdeveloped nationsdevelopmentaldietselderly miceepidemiologic dataepidemiologic investigationepidemiology studyextracellulareye disorderfat metabolismfirst in manfirst-in-humangene augmentation interventiongene augmentation therapygene complementationgene repair therapygene therapygene-based therapygene-based treatmentgene-directed therapygene-targeted therapygene-targeted treatmentgenetic analysisgenetic therapygenomic therapygeriatrichiPSChuman iPShuman iPSChuman induced pluripotent cellhuman induced pluripotent stem cellshuman inducible pluripotent stem cellshuman inducible stem cellsiPSiPSCiPSCsin vitro Assayin vivoin vivo Modelinduced human pluripotent stem cellsinduced pluripotent cellinduced pluripotent stem cellinducible pluripotent cellinducible pluripotent stem cellintravitreal injectionlipid metabolismmimeticsminimally invasivemodel of animalmouse modelmurine modelnovelocular diseaseocular disorderold miceophthalmopathypathophysiologypathwaypolymorphismpre-clinicalpre-clinical efficacypreclinicalpreclinical efficacypreclinical findingspreclinical informationpublic health relevancerAAVrecombinant AAVresponsereverse cholesterol transportrisk allelerisk generisk genotyperisk locirisk locusrisk variantsenile macular diseasesenior citizensocial rolesocio-economicsocio-economicallysocioeconomicallysocioeconomicssubretinal injectiontherapeutic targettraffickingtransgenetransgene expressiontreatment strategyvision lossvisual loss
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Gene therapy targeting CFH and lipoprotein dynamics in AMD
ABSTRACT

Age-related macular degeneration (AMD) is the most common cause of irreversible blindness among the

elderly in industrialized countries, and there are no treatments for the majority of patients. Early hallmarks of

AMD are the formation of lipid- and protein-rich, extracellular…

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Gene therapy targeting CFH and lipoprotein dynamics in AMD — DUKE UNIVERSITY | UNITED STATES | Sept 2025 | Dev Procure