grant
Pharmacological studies of RHO-associated retinitis pigmentosa
Organization UNIVERSITY OF PITTSBURGH AT PITTSBURGHLocation PITTSBURGH, UNITED STATESPosted 1 Aug 2020Deadline 28 Feb 2030
NIHUS FederalResearch GrantFY2026(hydroxymethylglutaryl-CoA reductase (NADPH)) kinase5'-AMP-activated protein kinaseAMP-activated kinaseAMP-activated protein kinaseAMPK enzymeAccelerationAcuteAdenosineAdenosine ReceptorsAffectAnabolismAnti-InflammatoriesAnti-Inflammatory AgentsAnti-inflammatoryAssayAtrophic ArthritisAutophagocytosisAutoregulationBioassayBiological AssayBiopsyBlindnessBlood PlasmaBody TissuesCancersCell BodyCell LineCellLineCellsChaperoneChronicClinical TrialsComplexCone PhotoreceptorsDNA mutationDataDiseaseDisorderDrug KineticsDrug ScreeningDrug TherapyDrug usageDrugsEnzyme GeneEnzymesExhibitsEye InfectionsFDA licensed drugsFDA-approved agentsFDA-approved drugFDA-approved medicationsFDA-approved pharmaceuticalsFDA-approved therapeutic agentFood and Drug Administration approved drugFood and Drug Administration approved medicationsFood and Drug Administration approved pharmaceuticalsFundingGenesGenetic ChangeGenetic defectGenetic mutationGoalsHMG CoA reductase (NADPH) kinaseHMG CoA reductase kinaseHMG coenzyme A reductase (NADPH) kinaseHPLCHereditaryHigh Performance Liquid ChromatographyHigh Pressure Liquid ChromatographyHigh Speed Liquid ChromatographyHomeostasisHumanImmune responseImmunomodulationInheritedInjectionsLeadLibrariesMalignant NeoplasmsMalignant TumorMeasuresMedicationMetabolicMethodsMethotrexateMethotrexate MethylaminopterinMethotrexatumMetotrexatoModern ManMolecularMolecular ChaperonesMutationNaIO3Neural RetinaOcular InfectionsOralOutcomeP1 PurinoceptorsPathogenesisPathway interactionsPatientsPb elementPersonsPharmaceutical PreparationsPharmacokineticsPharmacological StudyPharmacological TreatmentPharmacologyPharmacology StudyPharmacotherapyPhase 1/2 Clinical TrialPhase I/II Clinical TrialPhotoreceptor CellPhotoreceptorsPhotosensitive CellPhysiological HomeostasisPigmentary RetinopathyPlasmaPlasma SerumPreventative treatmentPreventive treatmentPurinergic P1 ReceptorsPurinesRegimenReticuloendothelial System, Serum, PlasmaRetinaRetina ProperRetinal ConeRetinal DegenerationRetinitis PigmentosaRheumatoid ArthritisRhodopsinRibonucleoside PhosphatesRibonucleotidesRiskRod PhotoreceptorsRouteSafetySightSignal PathwayStrains Cell LinesSynapsesSynapticSystemTapetoretinal DegenerationTherapeuticThickThicknessTimeTissuesToxic effectToxicitiesVisionVisual PurpleVisual ReceptorWorkaberrant folded proteinaberrant folded proteinsabnormal folded proteinabnormal folded proteinsautophagyautosomebiosynthesiscell immortalizationcompound repositioningcompound repurposingcone cellcultured cell linedegenerative retina diseasesdetermine efficacydrug candidatedrug discoverydrug interventiondrug repositioningdrug repurposingdrug treatmentdrug usedrug/agenteffective therapyeffective treatmentefficacy analysisefficacy assessmentefficacy determinationefficacy evaluationefficacy examinationefficacy studyevaluate efficacyexamine efficacyextracellulargenome mutationheavy metal Pbheavy metal leadhost responsehydroxymethylglutaryl-CoA-reductase kinaseimmune modulationimmune regulationimmune system responseimmunologic reactivity controlimmunomodulatoryimmunoregulationimmunoregulatoryimmunoresponseimprovedin vivoinhibitorintravenous injectionintravitreal injectionmalignancymetabolomemetabonomemisfolded proteinmisfolded proteinsmouse modelmurine modelmutantneoplasm/cancerneuralneuroprotectionneuroprotectivenew drug treatmentsnew drugsnew pharmacological therapeuticnew therapeutic uses for existing drugsnew therapeuticsnew therapynew use of drugnew uses for an approved drugnew uses for existing drugsnext generationnext generation therapeuticsnovelnovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel therapeuticsnovel therapypathwaypharmaceutical interventionpharmacologicpharmacological interventionpharmacological repurposingpharmacological therapypharmacology interventionpharmacology treatmentpharmacotherapeuticsphotoreceptor degenerationpolyglutamatepre-clinicalpre-clinical studypreclinicalpreclinical studyprotein homeostasisproteostasisproteotoxic proteinproteotoxinrepositioning approved drugsrepositioning existing drugsrepurpose approved drugsrepurpose approved medicationrepurpose approved therapeuticrepurpose existing drugsrepurpose existing medicationrepurpose existing medicinerepurpose existing therapeuticsrepurpose existing therapiesrepurpose medicinerepurposingrepurposing a drugrepurposing agentrepurposing candidatesrepurposing established drugsrepurposing established medicationrepurposing existing pharmacological agentsrepurposing medicationrepurposing of already existing drugsrepurposing pharmaceuticalsretina degenerationretinal degenerativeretinal degenerative diseasesretinal rodsrheumatic arthritisrod and cone dystrophyrod cellrod-cone dystrophysafety studyscreeningscreeningssodium iodatesubcutaneoussubdermalsuccesssynapsetherapeutic repositioningtherapeutic repurposingvision lossvisual functionvisual loss
Description preview
RHODOPSIN-associated autosomal dominant retinitis pigmentosa (RHO-adRP) affects 400,000-
600,000 people worldwide. Currently, there is no effective treatment for RHO-adRP. Our long-term
goal is to develop an effective and safe pharmacological treatment for RHO-adRP. This project
focuses on repurposing methotrexate (MTX), a drug used for cancer and…
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