grant
Clinical and mechanistic studies defining optimal preparative approaches to infants with IL2RG/JAK3/RAG1/RAG2 SCID: a randomized trial of busulfan dosage
Organization NATIONAL MARROW DONOR PROGRAMLocation MINNEAPOLIS, UNITED STATESPosted 13 Aug 2024Deadline 31 May 2029
NIHUS FederalResearch GrantFY20252 year old2 years of ageATGAMActive Follow-upAdoptive TransferAffectAllogenicAntibody ResponseAntigensAntithymocyte GlobulinAntithymoglobulinArea Under CurveB blood cellsB cellB cellsB-Cell DevelopmentB-CellsB-LymphocytesB-cellBiological MarkersBiologyBirthBloodBlood Precursor CellBlood Reticuloendothelial SystemBone Marrow GraftingBone Marrow TransplantBone Marrow TransplantationBussulfamBusulfanBusulfanumCD132CD19CD19 geneCOVID-19CV-19Cell CompartmentationCell CompartmentationsCell TherapyChemotherapy ProtocolChemotherapy RegimenChemotherapy-Oncologic ProcedureChildhoodChimerismClinicalClinical TrialsCombination Chemotherapy RegimenCoronavirus Infectious Disease 2019Cytokine Signal TransductionCytokine SignalingDNA TherapyDataDevelopmentDiseaseDisorderDomestic RabbitDoseElementsEngraftmentEnrollmentEnsureExhibitsFundingFutureGene Transfer ClinicalGeneticGenetic DiseasesGenetic InterventionGenotypeGoalsGraft RejectionGrantHSC transplantationHematopoieticHematopoietic Progenitor CellsHematopoietic Stem Cell TransplantHematopoietic Stem Cell TransplantationHematopoietic stem cellsHost vs Graft ReactionIGHIGH@ gene clusterIL21IL2RGIL2RG geneIMD4IgH locusImmuneImmune GlobulinsImmune ToleranceImmune systemImmunesImmunityImmunoglobulin Heavy Chain GenesImmunoglobulin Heavy GeneImmunoglobulin Heavy LocusImmunoglobulinsImmunologic ToleranceIn VitroInfantInfectionJAK-3JAK3JAK3 geneJAK3 proteinJAKLJanus kinase 3JointsLJAKLifeLymphatic cellLymphocyteLymphocyticMHC ReceptorMajor Histocompatibility Complex ReceptorMarrow TransplantationMature T-CellMature T-LymphocyteMeasuresMyelogenousMyeloidMyeloid CellsNeonatal ScreeningNewborn Infant ScreeningNorth AmericaOpportunistic InfectionsOrphan DiseaseOryctolagus cuniculusOutcomeOutputPK/PDParticipantParturitionPatientsPeripheralPharmacodynamicsProcessProgenitor Cell EngraftmentProgenitor CellsProphylactic treatmentProphylaxisProtocolProtocols documentationQuimioterapiaRabbitsRabbits MammalsRag1Rag1 MouseRandomization trialRandomizedRare DiseasesRare DisorderRegimenRegulationReplacement TherapyRiskSafetySamplingScienceSevere Combined ImmunodeficiencySevere Combined Immunodeficiency SyndromeSevere Combined Immunologic DeficiencySiblingsSourceStem Cell likeSubgroupSulfabutinSurvival RateSuspension substanceSuspensionsT cell reconstitutionT-Cell Antigen ReceptorsT-Cell DevelopmentT-Cell OntogenyT-Cell ReceptorT-CellsT-LymphocyteT-Lymphocyte DevelopmentTSPATestingTetanusTetanus VaccineTherapy trialThio-TepaThiofosfamideThiophosphamideThiophosphoramideThiotepaThymoglobulinThymusThymus GlandThymus ProperThymus Reticuloendothelial SystemToxic effectToxicitiesTransplant RejectionTransplantationTransplantation RejectionTriethylene thiophosphoramideTriethylenethiophosphoramideactive followupadaptive immune responseage 2 yearsaged 2 yearsaged two yearsarmbio-markersbiologic markerbiomarkerblood cell progenitorblood progenitorblood stem cellblood stem cell transplantationblood-forming stem cellcancer chemotherapycell based interventioncell mediated interventioncell mediated therapiescell-based therapeuticcell-based therapycellular therapeuticcellular therapyclinical outcome assessmentclostridial tetanuscohortcombined T and B cell inborn immunodeficiencyconditioningcoronavirus disease 2019coronavirus disease-19coronavirus infectious disease-19curative interventioncurative therapeuticcurative therapycurative treatmentsdesigndesigningdevelopmentaldosageefficacy testingenrollexhaustionfludarabinefollow upfollow-upfollowed upfollowupgene repair therapygene therapygene-based therapygenetic conditiongenetic disordergenetic therapygenomic therapyhematopoietic cell transplantationhematopoietic cellular transplantationhematopoietic progenitorhematopoietic progenitor cell transplantationhematopoietic stem progenitor cellhemopoietichemopoietic progenitorhemopoietic stem cellhigh riskhost versus graft reactionhost vs. graft reactionimmune reconstitutionimmune response to vaccinationimmune response to vaccinesimmune system toleranceimmune unresponsivenessimmunogenimmunoglobulin heavy chain locusimmunological paralysisimprovedinterleukin-21lymph cellnewborn screeningnovelorphan disorderpediatricpharmacokinetics and pharmacodynamicsprimary end pointprimary endpointprimary outcomeprogenitor capacityprogenitor cell likeprogenitor-likeprospectiverandomisationrandomizationrandomized trialrandomly assignedresponsestandard carestandard of carestandard treatmentstem cell characteristicsstem cell engraftmentstem cellsstem-likestemnesstargeted drug therapytargeted drug treatmentstargeted therapeutictargeted therapeutic agentstargeted therapytargeted treatmentthymus derived lymphocytetransplanttransplant therapytransplant treatmenttransplantation therapytransplantation treatmenttrial designtwo year oldtwo years of agevaccine associated immune responsevaccine immune responsevaccine immunogenicityvaccine induced immune responsevaccine responsevaccine responsivenessvaccine-induced responseyounger age
Description preview
Project Summary
Severe combined immunodeficiency (SCID) is a group of genetic disorders that abrogate T cell development
and function. Allogeneic hematopoietic cell transplantation (HCT) is the standard treatment for the disease and
can be performed successfully in SCID patients without the high dose pre-HCT busulfan conditioning due to the…
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