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Evaluation of Human Retinal Ganglion Cell Regenerative Potential
Organization JOHNS HOPKINS UNIVERSITYLocation BALTIMORE, UNITED STATESPosted 1 Dec 2024Deadline 31 May 2025 ⚠️
NIHUS FederalResearch GrantFY2025AblationAllelesAllelomorphsAreaAssayAxonBehaviorBehavioral AssayBioassayBiological AssayBlindnessBrachydanio rerioBrainBrain Nervous SystemCRISPR activationCRISPR activatorCRISPR approachCRISPR based activationCRISPR based approachCRISPR gene activationCRISPR interferenceCRISPR methodCRISPR methodologyCRISPR techniqueCRISPR technologyCRISPR toolsCRISPR transcription activationCRISPR transcriptional activationCRISPR-CAS-9CRISPR-Cas-9-mediated gene activationCRISPR-based gene activationCRISPR-based methodCRISPR-based techniqueCRISPR-based technologyCRISPR-based toolCRISPR-dCAS9 ActivatorCRISPR-dCas9-mediated repressionCRISPR-mediated transcriptional activationCRISPR/CAS approachCRISPR/CAS9 activationCRISPR/CAS9 gene activationCRISPR/Cas methodCRISPR/Cas technologyCRISPR/Cas9CRISPR/Cas9 technologyCRISPR/dCas9 activationCRISPR/dCas9 interferenceCRISPR/dCas9-based transcriptional activationCRISPR/dCas9-mediated transcriptional inhibitionCRISPRaCRISPRiCandidate Disease GeneCandidate GeneCas nuclease technologyCell BodyCell Communication and SignalingCell LineCell SignalingCell SurvivalCell TransplantationCell ViabilityCellLineCellsClinicClustered Regularly Interspaced Short Palindromic Repeats approachClustered Regularly Interspaced Short Palindromic Repeats interferenceClustered Regularly Interspaced Short Palindromic Repeats methodClustered Regularly Interspaced Short Palindromic Repeats methodologyClustered Regularly Interspaced Short Palindromic Repeats techniqueClustered Regularly Interspaced Short Palindromic Repeats technologyCranial Nerve IIDNA mutationDanio rerioDataData SetDendritesDevelopmentDiseaseDisorderEligibilityEligibility DeterminationEmbryoEmbryonicEncephalonEnvironmentEvaluationEyeEyeballFishesGeneralized GrowthGenesGeneticGenetic ChangeGenetic DiseasesGenetic ScreeningGenetic defectGenetic mutationGlaucomaGoalsGrowthHandHumanIn VitroIn vivo analysisIntracellular Communication and SignalingIntrinsic factorKnock-outKnockoutLiteratureMMAC1MMAC1 proteinMammaliaMammalsMiceMice MammalsMiningModern ManMurineMusMutated in Multiple Advanced Cancers 1MutationNatural regenerationNeuritesOptic NerveOuter pigmented layer of retinaPHTS genePHTS proteinPTENPTEN genePTEN proteinPTEN1PatientsPatternPhosphatase and Tensin HomologPhosphatase and Tensin Homolog Deleted on Chromosome 10Pigment cell layer of retinaPigmented layer of retinaPredictive FactorProgenitor CellsProtocol ScreeningProxyReaderRecovery of FunctionRegenerationRegenerative MedicineRegenerative capacityResearchResearch ResourcesResourcesRetinaRetinal Ganglion CellsRetinal Pigment EpitheliumRetinal pigment epithelial cellsSecond Cranial NerveSeriesSightSignal TransductionSignal Transduction SystemsSignalingStrains Cell LinesStructure of retinal pigment epitheliumTectum MesencephaliTemperatureTestingTherapeuticTimeTissue GrowthTransmissionTransplantationVisionVisualWorkZebra DanioZebra FishZebrafishactivating CRISPR technologyaxon regenerationaxonal regenerationbiological signal transductioncell regenerationcell typecellular regenerationcellular transplantconfocal imagingcultured cell linedevelopmentalendogenous progenitorendogenous stem cellsfunctional recoverygene functiongene testinggene-based testinggenetic conditiongenetic disordergenetic testinggenome mutationglaucomatoushandshuman progenitor cell derivedhuman stem cell-derivedimaging in vivoimprovedin vitro testingin vivo evaluationin vivo imagingin vivo testinginsightmesencephalic tectummidbrain tectummutated in multiple advanced cancers 1 proteinneuroprotectionneuroprotectiveontogenyoptic nerve regenerationoverexpressoverexpressionpermissivenessphosphatase and tensin homologue on chromosome tenprogenitor biologyprogenitor cell biologyrapid methodrapid techniqueregenerateregeneration abilityregeneration based therapyregeneration capacityregeneration potentialregeneration therapyregenerativeregenerative potentialregenerative therapeuticsregenerative therapyrepressing CRISPR-dCas9 systemrestorationrestore sightrestore visionretina transplantationretinal axonretinal ganglionretinal ganglion cell degenerationretinal imagingretinal regenerationretinal transplantationscRNA sequencingscRNA-seqscreeningscreeningssight restorationsingle cell RNA-seqsingle cell RNAseqsingle cell expression profilingsingle cell transcriptomic profilingsingle-cell RNA sequencingstem and progenitor biologystem cell biologystem cellssuccesstectaltectumtectum mesencephalicumtectum structuretime usetooltransfer learningtransmission processtransplantvision lossvision restorationvisual functionvisual loss
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PROJECT SUMMARY
Retinal ganglion cells (RGCs) are solely responsible for transmitting signals from the eye to the brain. They do
so via their axons, which make up the optic nerve (ON). As such, loss of RGCs results in diseases like glaucoma,
a leading cause of irreversible blindness worldwide. Loss of RGCs is considered irreversible, as the human…
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