grant
Synthetic introns for selective targeting of RNA splicing factor-mutant leukemia
Organization FRED HUTCHINSON CANCER CENTERLocation SEATTLE, UNITED STATESPosted 1 Aug 2023Deadline 31 Jul 2028
NIHUS FederalResearch GrantFY20253' Splice SiteAML - Acute Myeloid LeukemiaAcute Myeloblastic LeukemiaAcute Myelocytic LeukemiaAcute Myelogenous LeukemiaAddressAffectAllelesAllelomorphsAnatomic SitesAnatomic structuresAnatomyBiomedical EngineeringBiotechBiotechnologyBlood DiseasesBody TissuesBreast CancerBreast Cancer CellCancer BiologyCancer PatientCancersCell BodyCellsChronic Myelomonocytic LeukemiaClinicalDNA mutationDeoxypyrimidine KinaseDeoxythymidine KinaseDiseaseDisorderDissectionDrug DeliveryDrug Delivery SystemsDrugsDysmyelopoietic SyndromesElementsEngineeringEventExonsFDA approvedFrequenciesGanciclovirGancyclovirGene ExpressionGeneralized GrowthGenetic ChangeGenetic defectGenetic mutationGranulocytic LeukemiaGrowthHSVHealthHematologic CancerHematologic DiseasesHematologic MalignanciesHematologic NeoplasmsHematological DiseaseHematological DisorderHematological MalignanciesHematological NeoplasmsHematological TumorHematopoieticHematopoietic CancerHematopoietic Cell TumorHematopoietic MalignanciesHematopoietic NeoplasmsHematopoietic Neoplasms including LymphomasHematopoietic TumorHematopoietic and Lymphoid Cell NeoplasmHematopoietic and Lymphoid NeoplasmsHerpes Simplex VirusHerpes labialis VirusHeterograftHeterologous TransplantationIL-15IL15IL15 ProteinIn VitroIndividualInterleukin-15Interleukin-15 PrecursorIntervening SequencesIntronsLeftLesionLeukemic CellMGC9721Malignant Breast NeoplasmMalignant CellMalignant Hematologic NeoplasmMalignant Hematopoietic NeoplasmMalignant MelanomaMalignant NeoplasmsMalignant TumorMediatingMedicationMelanomaMelanoma CellMessenger RNAMethodsMolecularMutateMutationMyelocytic LeukemiaMyelodysplastic DiseaseMyelodysplastic SyndromesMyelogenous LeukemiaMyeloid DiseaseMyeloid LeukemiaMyeloid MalignancyMyeloid NeoplasmMyeloid TumorMyeloproliferative DisordersMyeloproliferative TumorsMyeloproliferative diseaseNatureNon-Lymphoblastic LeukemiaNon-Lymphocytic LeukemiaNonlymphoblastic LeukemiaNonlymphocytic LeukemiaNordeoxyguanosinePatient CarePatient Care DeliveryPatientsPatternPharmaceutical PreparationsPhysiciansPrecision therapeuticsProductionProteinsRNA SplicingRecurrenceRecurrentRefractoryRefractory Anemia with an Excess of BlastsRefractory anaemia with excess blastsSC35SRSF2SRSF2 geneSafetyScientistSerine/Arginine-Rich Splicing Factor 2SimplexvirusSmoldering LeukemiaSolid NeoplasmSolid TumorSplice Acceptor SitesSpliceosomesSplicingSystemTechnologyTestingTherapeuticThymidine KinaseTissue GrowthTissuesWorkXenograftXenograft procedureXenotransplantationacute granulocytic leukemiaacute myeloid leukemiabio-engineeredbio-engineersbioengineeringbiological engineeringblood cancerblood disorderbreast tumor cellcancer cellcancer of bloodcancer of the bloodcancer typecare for patientscare of patientscaring for patientscell killingcell typeclinical phenotypeconventional therapyconventional treatmentdetermine efficacydrug/agentefficacy analysisefficacy assessmentefficacy determinationefficacy evaluationefficacy examinationevaluate efficacyexamine efficacyexperimentexperimental researchexperimental studyexperimentsfunctional genomicsgenome mutationhemopoieticimmunostimulatory therapyimmunostimulatory treatmentin vivoinsightleukemialipid based nanoparticlelipid nanoparticleloss of functionmRNAmalignancymalignant breast tumormutantmyelodysplasiamyeloid granulocytic leukemiamyeloproliferative neoplasmmyelosisnative protein drugneoplasm relapseneoplasm/cancernew drug treatmentsnew drugsnew pharmacological therapeuticnew technologynew therapeuticsnew therapynext generation therapeuticsnovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel technologiesnovel therapeuticsnovel therapyontogenypharmaceutical proteinprecision therapiesprecision treatmentprotein drug agentprotein expressionprotein-based drugselective expressionselectively expressedside effectsuicide genetherapeutic proteintherapeutic targettumortumorigenicxeno-transplantxeno-transplantation
Description preview
SUMMARY
Many cancers carry recurrent, change-of-function mutations affecting RNA splicing factors, resulting in
sequence-specific changes in RNA splicing that promote disease initiation and progression. These
“spliceosomal mutations” are the most common class of mutations in myelodysplastic syndromes (MDS) and
related hematologic disorders, which…
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