grant
Metabolic regulation and manipulation of telomerase in hematopoiesis
Organization BOSTON CHILDREN'S HOSPITALLocation BOSTON, UNITED STATESPosted 1 Dec 2015Deadline 28 Feb 2031
NIHUS FederalResearch GrantFY2026AffectAwardAwarenessBiologyBlood CellsBlood DiseasesBlood Precursor CellBone marrow failureCardiovascular DiseasesCell BodyCell TherapyCellsCirrhosisClinical TrialsCole syndromeCole-Rauschkolb-Toomey syndromeDNA TherapyDNA mutationDefectDegenerative DisorderDiagnosisDiseaseDisorderDysfunctionDyskeratosis CongenitaDysmyelopoietic SyndromesEC 2.7.7.49Engman syndromeEnzyme GeneEnzymesEpigeneticEpigenetic ChangeEpigenetic MechanismEpigenetic ProcessFailureFunctional RNAFunctional disorderGWA studyGWASGene Transfer ClinicalGene variantGenesGenetic ChangeGenetic EngineeringGenetic Engineering BiotechnologyGenetic Engineering Molecular BiologyGenetic InterventionGenetic defectGenetic mutationGenetic studyGenotypeGoalsHealthHematologic Body SystemHematologic DiseasesHematologic Organ SystemHematological DiseaseHematological DisorderHematopoiesisHematopoieticHematopoietic Body SystemHematopoietic Cellular Control MechanismsHematopoietic Progenitor CellsHematopoietic SystemHematopoietic stem cellsHepatic CirrhosisHepatic DisorderHumanHuman EngineeringHuman GeneticsImpairmentIn VitroIntermediary MetabolismInterventionKnowledgeLengthLength of LifeLinkLiver CirrhosisLiver FibrosisLiver diseasesLongevityLung DiseasesLung Tissue FibrosisMapsMetabolicMetabolic ControlMetabolic ProcessesMetabolismMissionModern ManMolecularMolecular DiseaseMoonMutationMyelodysplastic DiseaseMyelodysplastic SyndromesNational Institutes of HealthNon-Polyadenylated RNANoncoding RNANontranslated RNANucleotide SynthesisNucleotidesOutcomePathologyPeripheral Blood CellPhysiopathologyPlayPrecision therapeuticsProcessProgenitor Cell TransplantationProgenitor CellsPublic HealthPulmonary DiseasesPulmonary DisorderPulmonary FibrosisPurines/Pyrimidines/Nucleotides/Nucleic Acids MetabolismRNARNA Gene ProductsRNA TranscriptaseRNA metabolismRNA-Dependent DNA PolymeraseRNA-Directed DNA PolymeraseRecombinant DNA TechnologyRefractory Anemia with an Excess of BlastsRefractory anaemia with excess blastsRegulationResearchReverse TranscriptaseRevertaseRibonucleic AcidRibonucleoproteinsRoleSmoldering LeukemiaStem Cell TransplantationStem cell transplantTelomeraseTelomerase RNA ComponentTelomere MaintenanceTestingTherapeuticThymidinThymidineTreatment EfficacyUnited States National Institutes of HealthUntranslated RNAVariantVariationWorkX-Linked Dyskeratosis CongenitaZinsser syndromeZinsser-Engman-Cole syndromeage associatedage correlatedage dependentage linkedage relatedage specificaging associatedaging associated diseaseaging associated disordersaging relatedaging related diseaseaging related disordersallelic variantblood cell formationblood cell progenitorblood disorderblood progenitorblood stem cellblood-forming stem cellcardiovascular disordercell based interventioncell mediated interventioncell mediated therapiescell typecell-based therapeuticcell-based therapycellular therapeuticcellular therapycirrhoticcongenital dyskeratosiscurative interventioncurative therapeuticcurative therapycurative treatmentsdegenerative conditiondegenerative diseasedevelop therapydisabilitydisease associated with agingdisease of agingdisease of the lungdisorder of agingdisorder of the lungdisorders associated with agingdisorders related to agingeffective therapyeffective treatmentengineered progenitor cellsengineered stem cellsepigeneticallyfibrosis in the lungfibrotic livergene repair therapygene therapygene-based therapygenetic therapygenetic variantgenetically engineeredgenome mutationgenome wide associationgenome wide association scangenome wide association studygenomewide association scangenomewide association studygenomic therapygenomic varianthematopoietic progenitorhematopoietic stem progenitor cellhemopoietichemopoietic progenitorhemopoietic stem cellhepatic diseasehepatic fibrosishepatopathyhuman progenitorhuman stem cellsin vivoinduced pluripotent stem cells derived from patientsinduced pluripotent stem cells from patientsinnovateinnovationinnovativeinsightintervention developmentintervention efficacyleukemialife spanlifespanliver disorderlung disorderlung fibrosismyelodysplasianew drug treatmentsnew drugsnew pharmacological therapeuticnew therapeuticsnew therapynext generation therapeuticsnoncodingnovelnovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel therapeuticsnovel therapynucleotide metabolismpathophysiologypatient derived human iPSpatient derived human iPSCpatient derived human induced pluripotent stem cellpatient derived iPSpatient derived iPSCpatient derived induced pluripotent cellspatient derived induced pluripotent stem cellspatient-derived pluripotent stem cellsprecision therapiesprecision treatmentprogenitor cell functionprogenitor cell geneprogenitor cell homeostasisprogenitor cell regenerationprogenitor cell self renewalprogenitor functionprogenitor geneprogenitor regenerationprogenitor self renewalprogenitor transplantationresponseresponse to therapyresponse to treatmentself-renewself-renewalsmall moleculesocial rolestem and progenitor cell functionstem and progenitor cell regenerationstem and progenitor cell self renewalstem and progenitor cell transplantationsstem and progenitor functionstem cell functionstem cell genesstem cell homeostasisstem cell regenerationstem cell self renewalstem cellstelomerase RNAtelomeretherapeutic candidatetherapeutic efficacytherapeutic responsetherapy developmenttherapy efficacytherapy responsetooltreatment developmenttreatment responsetreatment responsivenesswhole genome association analysiswhole genome association study
Description preview
ABSTRACT
Telomerase plays a vital role in human health and longevity, yet its regulation in human cells remains poorly
understood. This gap in knowledge limits the ability to develop therapies for a growing spectrum of telomere
biology disorders (TBDs) linked to telomerase dysfunction. The long-term goal of this project is to enable
therapeutic…
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