grant
Targeting Regulatory T cells for Factor VIII Tolerance
Organization STANFORD UNIVERSITYLocation STANFORD, UNITED STATESPosted 20 Sept 2025Deadline 30 Jun 2030
NIHUS FederalResearch GrantFY20250-11 years old21+ years oldATP-protein phosphotransferaseAdultAdult HumanAdverse effectsAffectAgeAntibodiesAntigensAntihemophilic FactorAutoimmune DiseasesAutoimmune StatusAutoimmunityB blood cellsB cellB cellsB-Cell ActivationB-CellsB-LymphocytesB-cellBiologic ModelsBiological MarkersBiological ModelsBleedingBlood Coagulation DisordersBlood Coagulation Factor VIIIBlood Plasma CellBlood erythrocyteBypassCD4 CellsCD4 Positive T LymphocytesCD4 T cellsCD4 helper T cellCD4 lymphocyteCD4+ T-LymphocyteCD4-Positive LymphocytesCell BodyCell Communication and SignalingCell IsolationCell SegregationCell SeparationCell Separation TechnologyCell SignalingCellsChildChild YouthChildhoodChildren (0-21)Chronic Kidney FailureChronic Renal DiseaseChronic Renal FailureClassificationClottingCoagulationCoagulation DisorderCoagulation Factor VIIICoagulation Factor VIIIcCoagulation ProcessCoagulopathyComplicationDevelopmentDiseaseDisorderDysfunctionECSFEPO-RElementsEndocrine Gland SecretionEpoetinErythrocytesErythrocyticErythropoiesisErythropoietinErythropoietin ReceptorExhibitsFK506 Binding Protein 12-Rapamycin Associated Protein 1FKBP12 Rapamycin Complex Associated Protein 1FRAP1FRAP1 geneFRAP2Factor VIIIFactor VIII DeficiencyFactor VIII F8BFailureFrequenciesFunctional disorderGenotypeGerminal CenterGoalsGrafting ProcedureHelper CellsHelper T-CellsHelper T-LymphocytesHelper-Inducer T-CellsHelper-Inducer T-LymphocyteHematologyHemophiliaHemophilia AHemorrhageHemostasisHemostatic AgentsHemostatic functionHemostaticsHereditaryHormonesHumanIV InfusionImmuneImmune ToleranceImmune responseImmunesImmunityImmunologic ToleranceImmunomodulationImmunosuppressionImmunosuppression EffectImmunosuppressive EffectIn VitroIndividualInducer CellsInducer T-LymphocytesInduction TherapyInfusionInfusion proceduresInheritedIntracellular Communication and SignalingIntravenous infusion proceduresInvestigationInvestigatorsKidneyKidney Urinary SystemKinase Family GeneLEF Transcription FactorLifeLymphoid Enhancer FactorMarrow erythrocyteMechanistic Target of RapamycinMediatingMediatorMiceMice MammalsModel SystemModelingModern ManMorbidityMorbidity - disease rateMurineMusNEOADJNeoadjuvantNeoadjuvant TherapyNeoadjuvant TreatmentOrgan TransplantationOrgan TransplantsOrganoidsPBMCPathologyPeripheral Blood Mononuclear CellPersonsPhenotypePhysiopathologyPlasma CellsPlasmacytesPopulationPreventionProcessProcoagulant ComponentProductionProtein InhibitionProtein KinaseProteinsQOLQuality of lifeRAFT1RapamuneRapamycinRecombinant EPORecombinant ErythropoietinRed Blood CellsRed CellRegulatory T-LymphocyteReplacement TherapyResearchResearch PersonnelResearchersRiskSafetySignal PathwaySignal TransductionSignal Transduction SystemsSignalingSirolimusSiteSolidSpleenSpleen Reticuloendothelial SystemStructure of germinal center of lymph nodeSurgical ManagementSystematicsT Cell FactorT cell responseT-Cell SubsetsT-CellsT-LymphocyteT-Lymphocyte SubsetsT4 CellsT4 LymphocytesTCF Transcription FactorTeff cellTestingTherapeuticTherapeutic HormoneThromboplastinogenToxic effectToxicitiesTraumaTregWorkactivated B cellsadaptive immune responseadaptive immunityadulthoodagesantihemophilic factor Aautoimmune conditionautoimmune disorderautoimmunity diseasebio-markersbiologic markerbiological signal transductionbiomarkerbleeding disorderblood corpusclesblood losscell sortingcentral tolerancechild patientschronic kidney diseaseclotting disordercohortcomplex Blood-coagulation factor VIIIdevelop therapydevelopmentaleffector T cellerythrocyte colony stimulating factorerythroid developmentexperienceglycogen synthase a kinasehematopoietinhost responsehuman modelhydroxyalkyl protein kinaseimmune modulationimmune regulationimmune suppressionimmune suppressive activityimmune suppressive functionimmune system responseimmune system toleranceimmune unresponsivenessimmunogenimmunologic reactivity controlimmunological paralysisimmunomodulatoryimmunoregulationimmunoregulatoryimmunoresponseimmunosuppressive activityimmunosuppressive functionimmunosuppressive responsein vivoinduction therapiesineffective therapiesineffective treatmentinfusionsinhibit proteininhibit proteinsinhibitorinhibitor druginhibitor therapeuticinhibitor therapyinnovateinnovationinnovativeintervention developmentintravenous infusionkidsmTORmammalian target of rapamycinmodel of humanmortalitymouse modelmurine modelneutralizing antibodynovelorgan allograftorgan graftorgan xenograftpathophysiologypediatricpediatric patientsperipheral tolerancepharmacologicphosphorylase b kinase kinaseplasmocyteplatelet cofactor Ipreventpreventingprotein inhibitionsregulatory T-cellsrenalresponsesuccesstherapy developmentthromboplastinogen Athymus derived lymphocytetransgene deliverytreatment developmentyoungster
Description preview
PROJECT SUMMARY
Children and adults with the inherited bleeding disorder hemophilia A are reliant on infusions of coagulation
protein factor VIII (FVIII) to treat bleeding episodes. The most significant complication of FVIII replacement
therapy is the formation of neutralizing antibodies called inhibitors against the infused FVIII protein.…
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