grant
Study of tyrosine kinase inhibitors as preventive therapy in RUNX1-FPD
Organization UNIV OF MASSACHUSETTS MED SCH WORCESTERLocation WORCESTER, UNITED STATESPosted 1 May 2025Deadline 30 Apr 2030
NIHUS FederalResearch GrantFY2026AML - Acute Myeloid LeukemiaAML1AMLCR1AbbreviationsAcuteAcute Lymphoblastic LeukemiaAcute Lymphocytic LeukemiaAcute Lymphoid LeukemiaAcute Myeloblastic LeukemiaAcute Myelocytic LeukemiaAcute Myelogenous LeukemiaAffectAntimorphic mutationApplications GrantsAutomobile DrivingBasal Transcription FactorBasal transcription factor genesBasic ResearchBasic ScienceBleedingBlood DiseasesBlood NeutrophilBlood PlateletsBlood Polymorphonuclear NeutrophilBlood Precursor CellBone MarrowBone Marrow Reticuloendothelial SystemBruiseC-KIT GeneCBFA2CD117CD117 AntigensCancer PatientCell BodyCellsChronic LeukemiaClinical ResearchClinical StudyClonal ExpansionClonal expansion of hematopoietic cellsClonal expansion of hematopoietic stem cellsClonal hematopoietic expansionCollectionContusionsDNA DamageDNA Damage RepairDNA InjuryDNA RepairDNA mutationDasatinibDataDephosphorylationDevelopmentDiseaseDisorderDominant NegativeDominant-Negative MutantDominant-Negative MutationDrug resistanceDrugsDysfunctionDysmyelopoietic SyndromesEPH- and ELK-Related Tyrosine KinaseEPH-and ELK-Related KinaseEphrin Type-A Receptor 8Ephrin Type-A Receptor 8 PrecursorFamilial Platelet DisorderFunctional disorderGametesGeneral Transcription Factor GeneGeneral Transcription FactorsGenesGenetic ChangeGenetic defectGenetic mutationGerm CellsGerm LinesGerm-Line CellsGerm-Line MutationGrant ProposalsHematologic CancerHematologic DiseasesHematologic MalignanciesHematologic NeoplasmsHematological DiseaseHematological DisorderHematological MalignanciesHematological NeoplasmsHematological TumorHematologyHematopoieticHematopoietic CancerHematopoietic Cell TumorHematopoietic MalignanciesHematopoietic NeoplasmsHematopoietic Neoplasms including LymphomasHematopoietic Progenitor CellsHematopoietic TumorHematopoietic and Lymphoid Cell NeoplasmHematopoietic and Lymphoid NeoplasmsHematopoietic stem cellsHemorrhageHereditaryHereditary MutationHumanHypersensitivityImatinibImmune systemIn VitroIndividualInflammationInflammatoryInheritedLifeMalignant Hematologic NeoplasmMalignant Hematopoietic NeoplasmMarrow NeutrophilMarrow plateletMast Cell Growth Factor ReceptorMediatingMedicationMiceMice MammalsModern ManMolecularMolecular AnalysisMultiple MyelomaMurineMusMutateMutationMyelodysplastic DiseaseMyelodysplastic SyndromesMyelogenousMyeloidNeutrophilic GranulocyteNeutrophilic LeukocyteNon-Receptor Type 11 Protein Tyrosine PhosphataseOnset of illnessPEBP2A2PEBP2aBPTK InhibitorsPTP-2 enzymePTP2CPTPN11PTPN11 genePathologyPathway interactionsPatientsPharmaceutical PreparationsPhase 2 Clinical TrialsPhase II Clinical TrialsPhenotypePhysiopathologyPlasma-Cell MyelomaPlateletsPolymorphonuclear CellPolymorphonuclear LeukocytesPolymorphonuclear NeutrophilsPrecancerous CellsPrecursor Cell Lymphoblastic LeukemiaPrecursor Lymphoblastic LeukemiaPredispositionPreleukemiaPremalignant CellPreventative therapyPreventative treatmentPreventive therapyPreventive treatmentProductionProgenitor Cell TransplantationProgenitor CellsProtein DephosphorylationProtein Tyrosine KinaseProtein Tyrosine Kinase EEKProtein Tyrosine Kinase InhibitorsProtein Tyrosine Phosphatase 2CProtein-Tyrosine Phosphatase 2CProto-Oncogene Protein c-kitPublishingRUNX1RUNX1 geneRecoveryRefractory Anemia with an Excess of BlastsRefractory anaemia with excess blastsRegulatory ElementReproductive CellsRiskRoleSCF ReceptorSCF Receptor GeneSCFRSHP2SHP2 PhosphataseSHPTP2Sex CellShp-2 tyrosine phosphataseSmoldering LeukemiaSomatic MutationStem Cell Factor ReceptorStem Cell Factor Receptor GeneStem Cell TransplantationStem cell transplantSusceptibilityTK InhibitorsTestingTherapeuticThrombocytesThrombocytopeniaThrombopeniaTranscription Factor Proto-OncogeneTranscription factor genesTyrosineTyrosine KinaseTyrosine Kinase InhibitorTyrosine Phosphatase SHP2Tyrosine-Protein Kinase Receptor EEKTyrosine-Specific Protein KinaseTyrosylprotein KinaseUnscheduled DNA Synthesisacute granulocytic leukemiaacute lymphatic leukemiaacute lymphogenous leukemiaacute lymphomatic leukemiaacute myeloid leukemiablood cancerblood cell progenitorblood disorderblood lossblood progenitorblood stem cellblood-forming stem cellc kitc-kit Proteinc-kit Receptorcancer of bloodcancer of the bloodcancer riskchemotherapyclonal expansions in the bloodclonal hematopoiesisclones in hematopoietic cellscytokinedetermine efficacydevelopmentaldisease onsetdisorder onsetdrivingdrug resistantdrug/agentefficacy analysisefficacy assessmentefficacy determinationefficacy evaluationefficacy examinationefficacy studyevaluate efficacyexamine efficacygenome mutationgerm-line defectgermline varianthematopoietic cell cloneshematopoietic differentiationhematopoietic progenitorhematopoietic stem cell clonalityhematopoietic stem progenitor cellhemopoietichemopoietic progenitorhemopoietic stem cellhigh riskhydroxyaryl protein kinaseiPSiPSCiPSCsinduced pluripotent cellinduced pluripotent stem cellinducible pluripotent cellinducible pluripotent stem cellinitial cellinsightintervention designkit Proto-Oncogene Proteinlife-time risklifetime riskmutantmyelodysplasiamyelomamyelomatosisneutrophilp145(c-kit)p145c-kitpathophysiologypathwayphase II protocolpreventpreventingprogenitorprogenitor transplantationresistance to Drugresistant to Drugresponsesexual cellsocial rolesomatic variantstem and progenitor cell transplantationsstem cellstherapy designtranscription factortreatment designtyrosyl protein kinase
Description preview
Abstract
Familial platelet disorder (FPD) is a rare, inherited, dominant disease that is associated with high lifetime-risk
of developing hematologic malignancies. FPD patients can live for decades in a “pre-leukemic” period before
disease onset, opening a therapeutic opportunity for the development of preventive treatments. Molecularly,
FPD is…
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