grant
Determining the Mechanism of Rhabdomyosarcoma Transformation from a Non-Myogenic Cell of Origin
Organization ST. JUDE CHILDREN'S RESEARCH HOSPITAL GRADUATE SCHOOL OF BIOMEDICAL SCIENCES, LLCLocation MEMPHIS, UNITED STATESPosted 13 May 2025Deadline 12 May 2028
NIHUS FederalResearch GrantFY202521+ years oldAdipose tissueAdultAdult HumanAutomobile DrivingBasal Transcription FactorBasal transcription factor genesBile TractBiliary SystemBiliary TreeBiologyBladderBladder Urinary SystemBody RegionsCRISPRCRISPR editing screenCRISPR screenCRISPR-based screenCRISPR/Cas systemCRISPR/Cas9 screenCUT&RUNCancer BiologyCell BodyCell Communication and SignalingCell SignalingCellsChildhood Soft Tissue SarcomaChromatinChromatin StructureCleavage Targets and Release Using NucleaseCleavage Under Targets and Release Using NucleaseClinicalClinical TrialsClustered Regularly Interspaced Short Palindromic RepeatsCommon NeoplasmCommon TumorConceptionsDNA RecombinationDataDevelopmentDevelopmental BiologyDiagnosisDiseaseDisorderEmbryoEmbryo DevelopmentEmbryogenesisEmbryonicEmbryonic DevelopmentEndothelial CellsEndotheliumEpigeneticEpigenetic ChangeEpigenetic MechanismEpigenetic ProcessEventExperimental DesignsExperimental ModelsExpression SignatureFDA approvedFaceFatty TissueGene Expression ProfileGene TranscriptionGeneral Transcription Factor GeneGeneral Transcription FactorsGenesGenetic RecombinationGenetic TranscriptionHead and NeckHead and neck structureHedgehog (Hh) signal transduction pathwayHi-CHistologicHistologicallyHistologyIn VitroIntracellular Communication and SignalingKO miceKnock-outKnock-out MiceKnockoutKnockout MiceKnowledgeMediatingMiceMice MammalsModelingMurineMusMuscleMuscle DevelopmentMuscle TissueMuscular DevelopmentMyogenic Regulatory FactorsNull MouseOncogenicOperative ProceduresOperative Surgical ProceduresOutcomePatientsPediatric Soft Tissue SarcomaProliferatingProstateProstate GlandProstatic GlandProteinsRNA ExpressionRadiationRecombinationRegional AnatomyResearchRhabdomyosarcomaSHHSHH geneSignal TransductionSignal Transduction SystemsSignalingSiteSkeletal MuscleSolid NeoplasmSolid TumorSonic HedgehogSonic Hedgehog (Shh) PathwaySonic Hedgehog PathwaySpecific qualifier valueSpecifiedSurgicalSurgical InterventionsSurgical ProcedureSurvival RateTestingTimeTrainingTranscriptionTranscription Factor Proto-OncogeneTranscription factor genesUnited StatesVoluntary Muscleadiposeadulthoodbiliary tractbiological signal transductionchemotherapyclustered regularly interspaced short palindromic repeats screenconditional knock-outconditional knockoutdevelopmentaldirected differentiationdrivingendothelial progenitorendothelial progenitor cellendothelial stem cellepigeneticallyexperimentexperimental researchexperimental studyexperimentsfacesfacialgene expression patterngene expression signaturehedgehog signalinghedgehog signaling pathwayhh signaling pathwayhigh riskimprovedin vitro Modelin vivoinsightinterstitialmouse modelmurine modelmuscle progenitormuscle progenitor cellmuscle stem cellmuscularnew drug targetnew druggable targetnew pharmacotherapy targetnew therapeutic targetnew therapy targetnovelnovel drug targetnovel druggable targetnovel pharmacotherapy targetnovel therapeutic targetnovel therapy targetprogenitorprogramspromoterpromotorscATAC sequencingscATAC-seqsingle cell ATAC-seqsingle cell ATAC-sequencingsingle cell Assay for Transposase Accessible Chromatin sequencingsingle cell sequencing assay for transposase accessible chromatinsingle-cell Assay for Transposase-Accessible Chromatin with sequencingsingle-cell assay for transposase-accessible chromatin using sequencingsingle-cell assay for transposase-accessible chromatin-seqskeletal muscle differentiationsmoothened signaling pathwaysurgerytargeted drug therapytargeted drug treatmentstargeted therapeutictargeted therapeutic agentstargeted therapytargeted treatmenttherapeutic targettranscription factortranscriptional profiletranscriptional signaturetumorurinary bladderwhite adipose tissueyellow adipose tissue
Description preview
Project Summary/Abstract
Rhabdomyosarcoma (RMS) is an aggressive solid tumor and the most common pediatric soft tissue sarcoma.
RMS treatment consists of chemotherapy, radiation, and surgery with no FDA approved targeted therapies to
date. Despite decades of research and clinical trials, high risk patients face an overall survival rate of less…
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