grant

Elucidating the molecular mechanisms of secondary cone degeneration in models of Retinitis Pigmentosa

Organization MASSACHUSETTS EYE AND EAR INFIRMARYLocation BOSTON, UNITED STATESPosted 1 Apr 2026Deadline 31 Mar 2031
NIHUS FederalResearch GrantFY2026ATRAAnti-VEGFAnti-VEGF Humanized Monoclonal AntibodyAnti-VEGF RhuMAbAssayBioassayBiological AssayBlindnessBlood VesselsBlood capillariesBlood-Retinal BarrierCandidate Disease GeneCandidate GeneCell BodyCell Communication and SignalingCell SignalingCellsCessation of lifeColor VisionsConeCone PhotoreceptorsDNA TherapyDNA mutationDataDeathDiseaseDisorderElectroporationElectroretinographyEventExtravasationFe elementGene Expression MonitoringGene Expression Pattern AnalysisGene Expression ProfilingGene Transfer ClinicalGenesGeneticGenetic ChangeGenetic InterventionGenetic defectGenetic mutationHereditaryHereditary DiseaseHeterogeneityHumanImmuneImmunesImpairmentIn vivo analysisInborn Genetic DiseasesInfiltrationInheritedInherited disorderIntracellular Communication and SignalingIronKO miceKnock-out MiceKnockout MiceLeakageMediatingMethodsMiceMice MammalsMoAb VEGFModelingModern ManMolecularMonoclonal Antibody Anti-VEGFMuller gliaMuller's cellMurineMusMutateMutationMüller cellMüller gliaNatureNull MousePatientsPersonsPhenocopyPhenotypePigmentary RetinopathyQOLQuality of lifeRecombinant Humanized Anti-VEGF Monoclonal AntibodyRecombinant Humanized Monoclonal Antibody to Vascular Endothelial Growth FactorRetinaRetinal ConeRetinal DiseasesRetinal DisorderRetinitis PigmentosaRetinoic AcidRhuMAb VEGFRodRod PhotoreceptorsSecondary toSightSignal PathwaySignal TransductionSignal Transduction SystemsSignalingSpillageTapetoretinal DegenerationTestingTherapeuticTrans Vitamin A AcidTranscript Expression AnalysesTranscript Expression AnalysisTretinoinTretinoinumVEGFVEGFsVP 16VP16Vascular Endothelial Growth FactorsVascularizationVisionVisual AcuityVitamin A AcidWorkall-trans-Retinoic Acidall-trans-Vitamin A acidanalyze gene expressionbevacizumabbiological signal transductioncapillarycell typeclinical translationclinically translatableconditional knock-outconditional knockoutcone cellcytokineelectroporative deliveryelectroretinogramgain of functiongene electrotransfergene expression analysisgene expression assaygene functiongene repair therapygene therapygene-based therapygenetic therapygenome mutationgenomic therapygenotyped patientshereditary disorderheritable disorderimprovedin vivoin vivo evaluationin vivo testinginborn errorinherited diseasesinherited genetic diseaseinherited genetic disorderloss of functionmouse modelmurine modelnoveloverexpressoverexpressionphotoreceptor degenerationpreservationpreventpreventingresponseretina diseaseretina disorderretinal rodsretinopathyrhorhuMabVEGFrod and cone dystrophyrod cellrod-cone dystrophyscreeningscreeningstrans-Retinoic Acidtranscriptional profilingvascularvectorvision lossvisual functionvisual loss
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Project Summary
Retinitis Pigmentosa (RP) is an inherited retinal disease afflicting 1 in 4,000 people worldwide. The disease

progresses initially by rod photoreceptor degeneration caused by mutations in rod-specific genes, although

different mutations in different genes converge upon the same rod degeneration phenotype in this disease.

However,…

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