grant

Investigating and targeting the translational landscape of DBA

Organization STANFORD UNIVERSITYLocation STANFORD, UNITED STATESPosted 1 Aug 2024Deadline 31 May 2029
NIHUS FederalResearch GrantFY20251, 2-Dehydrocortisone4E-BP15' Untranslated Regions5'UTRAddressAdrenal Cortex HormonesAntioncogene Protein p53ApoptosisApoptosis PathwayBasal Transcription FactorBasal transcription factor genesBiogenesisBirth DefectsBody TissuesBone marrow failureCell BodyCell CountCell Cycle ArrestCell NumberCellsCellular Tumor Antigen P53CharacteristicsChemicalsCongenital AbnormalityCongenital Anatomical AbnormalityCongenital DefectsCongenital DeformityCongenital MalformationCorticoidsCorticosteroidsCraniofacial AbnormalitiesDNA HelicasesDNA Unwinding ProteinsDNA mutationDNA unwinding enzymeDataDefectDehydrocortisoneDelta(1)-CortisoneDeltacortisoneDeltadehydrocortisoneDevelopmentDiamond-Blackfan anemiaDigitDigit structureDiseaseDisorderDrug TherapyDysfunctionEIF4EBP1EIF4EBP1 geneElementsErythroidExtremitiesFDA approvedFailureFunctional disorderGene ExpressionGeneral Transcription Factor GeneGeneral Transcription FactorsGeneticGenetic ChangeGenetic defectGenetic mutationHematologyHematopoieticHigh Throughput AssayHumanImpairmentKnowledgeLeadLimb structureLimbsLinkMapsMediatingMessenger RNAMetacortandracinMethodologyModern ManMutationNon-Polyadenylated RNANon-TrunkOncoprotein p53Origin of LifeP53PathogenesisPathologicPathway interactionsPb elementPharmacological TreatmentPharmacotherapyPhenotypePhosphoprotein P53Phosphoprotein pp53PhysiopathologyPlayPrednisonePrednisonumProgrammed Cell DeathProtein BiosynthesisProtein TP53RNARNA Gene ProductsRibo-seqRibonucleic AcidRibosomal Peptide BiosynthesisRibosomal Protein BiosynthesisRibosomal Protein SynthesisRibosomal ProteinsRibosomesRoleSortingSpecificityStressStructureTP53TP53 geneTRP53TechnologyTestingTherapeuticTimeTissuesTranscriptTranscription Factor Proto-OncogeneTranscription factor genesTranslatingTranslationsTumor Protein p53Tumor Protein p53 Genecell typeclinical phenotypeclinical relevanceclinically relevantcraniofacialcraniofacial anomaliescraniofacial defectscraniofacial malformationcraniofaciesdelta-Cortisonedevelopmentaldisease phenotypedrug interventiondrug treatmenterythroid differentiationexperimentexperimental researchexperimental studyexperimentsgain of functiongenome mutationgenome wide analysisgenome wide studiesgenome-wide analysisgenome-wide identificationheavy metal Pbheavy metal leadhelicasehemopoietichigh throughput screeninghuman diseasein vivoloss of functionmRNAmRNA Leader Sequencesmouse modelmurine modelnew drug targetnew drug treatmentsnew druggable targetnew drugsnew pharmacological therapeuticnew pharmacotherapy targetnew technologynew therapeutic targetnew therapeuticsnew therapynew therapy targetnext generation therapeuticsnovelnovel drug targetnovel drug treatmentsnovel druggable targetnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel pharmacotherapy targetnovel technologiesnovel therapeutic targetnovel therapeuticsnovel therapynovel therapy targetp53 Antigenp53 Genesp53 Tumor Suppressorpathophysiologypathwaypharmaceutical interventionpharmacological interventionpharmacological therapypharmacology interventionpharmacology treatmentpharmacotherapeuticsprotein p53protein synthesisribosome footprint profilingribosome profilingribosomopathysmall moleculesocial roletranscription factortranslationtranslation factor
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Description preview

In ribosomopathies, perturbed expression of ribosome components leads to tissue-specific phenotypes, such
as limb and craniofacial defects as well as bone marrow failure. A key example of a ribosomopathy is Diamond

Blackfan Anemia (DBA) which results in an erythroid-specific disease manifestation. What accounts for such

tissue-selective…

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Investigating and targeting the translational landscape of DBA — STANFORD UNIVERSITY | UNITED STATES | Aug 2024 | Dev Procure