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High-resolution functional mapping of non-coding sequences regulating fetal hemoglobin

Organization HARVARD MEDICAL SCHOOLLocation BOSTON, UNITED STATESPosted 1 Mar 2025Deadline 28 Feb 2026 ⚠️
NIHUS FederalResearch GrantFY202521+ years oldAddressAdultAdult HumanAllelesAllelomorphsAssayB-thalassemiaBasal Transcription FactorBasal transcription factor genesBindingBinding SitesBioassayBiologicalBiological AssayCD19CD19 geneCRISPRCRISPR approachCRISPR based approachCRISPR methodCRISPR methodologyCRISPR techniqueCRISPR technologyCRISPR toolsCRISPR-CAS-9CRISPR-based methodCRISPR-based techniqueCRISPR-based technologyCRISPR-based toolCRISPR/CAS approachCRISPR/Cas methodCRISPR/Cas systemCRISPR/Cas technologyCRISPR/Cas9CRISPR/Cas9 technologyCas nuclease technologyCell LineCellLineChIP assayClustered Regularly Interspaced Short Palindromic RepeatsClustered Regularly Interspaced Short Palindromic Repeats approachClustered Regularly Interspaced Short Palindromic Repeats methodClustered Regularly Interspaced Short Palindromic Repeats methodologyClustered Regularly Interspaced Short Palindromic Repeats techniqueClustered Regularly Interspaced Short Palindromic Repeats technologyCombining SiteDNA BindingDNA Binding InteractionDNA TherapyDNA boundDataDevelopmentERYF1ElementsEnhancersErythroidErythroid Precursor CellsErythroid Progenitor CellsErythroid Stem CellsErythropoietic Progenitor CellsErythropoietic Stem CellsFDA approvedFetal HbFetal HemoglobinFunctional RNAGATA Binding Protein 1GATA-1GATA1GATA1 geneGATA1 proteinGATA1 transcription factorGF-1 proteinGene Transfer ClinicalGeneral Transcription Factor GeneGeneral Transcription FactorsGenesGenetic InterventionGenetic Population StudyGenetics-MutagenesisGuide RNAHaplotypesHb SS diseaseHbFHbSS diseaseHemoglobinHemoglobin FHemoglobin S DiseaseHemoglobin sickle cell diseaseHemoglobin sickle cell disorderHemoglobinopathiesIn SituLengthLinkLogicMapsMeasuresModelingMolecular InteractionMutagenesisMutagenesis Molecular BiologyNF-E1 erythroid-specific transcription factorNFE1 proteinNoncoding RNANontranslated RNANucleic Acid Regulator RegionsNucleic Acid Regulatory SequencesOutcomePatternPhenotypePopulationProbabilistic ModelsProbability ModelsRNA SeqRNA sequencingRNAseqReactive SiteRegulationRegulatory ElementRegulatory RegionsReporterResearchResolutionSCL Transcription FactorSickle Cell AnemiaSingle Base PolymorphismSingle Nucleotide PolymorphismSortingStatistical ModelsStem Cell Leukemia Hematopoietic Transcription FactorStem Cell ProteinStrains Cell LinesT-Cell Acute Lymphocytic Leukemia 1 GeneT-cell acute lymphocytic leukemia 1 proteinTAL1TAL1 Gene ProductTAL1 geneTCL5TherapeuticTranscription Factor GATA1Transcription Factor Proto-OncogeneTranscription factor genesTranscriptional ControlTranscriptional RegulationTranslatingUntranslated RNAVariantVariationWorkadulthoodbasebase editingbasesbeta Thalassemiabiologicchromatin immunoprecipitationcultured cell linedevelopmentaleffective therapyeffective treatmenterythroid progenitorerythroid-committed progenitorsfetalfetal form of hemoglobinfetal globinflexibilityflexiblegRNAgamma Globingene repair therapygene therapygene-based therapygenetic regulatory elementgenetic therapygenome editinggenomic editinggenomic therapyglobin transcription factor 1indelinsertion/deletioninsertion/deletion mutationinsightnew drug treatmentsnew drugsnew pharmacological therapeuticnew therapeuticsnew therapynext generation therapeuticsnoncodingnovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel therapeuticsnovel therapynuclear factor-erythroid 1nucleasep-Thalassemiapreventpreventingpromoterpromotorresolutionssensorsickle cell diseasesickle cell disordersickle diseasesicklemiasingle nucleotide variantstatistical linear mixed modelsstatistical linear modelstranscription factortranscription factor NFE-1transcriptome sequencingtranscriptomic sequencingβ-thalassemiaγ-Globin

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Description preview

High-resolution functional mapping of non-coding sequences regulating fetal hemoglobin
Transcriptional regulation from non-coding sequences and interacting DNA-binding transcription factors (TF)

govern the developmental switch from fetal to adult hemoglobin, commonly referred to as “hemoglobin

switching”. The reinduction of fetal hemoglobin (HbF)…

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