grant
iPS-technology and patient specific disease models
Organization NATIONAL HEART, LUNG, AND BLOOD INSTITUTELocation UNITED STATES
NIHUS FederalResearch GrantFY2025ADA-SCIDAPRF proteinAcademyAcute-Phase Response FactorAddressAdenosineAdenosine A(2B) ReceptorAdenosine A2B ReceptorAdenosine AminohydrolaseAdherent CultureAffectAlimentary CanalAngiitisAnimal ModelAnimal Models and Related StudiesApoplexyArterial DisorderArteriesArteriopathyAssayAtrophicAtrophyBasal Transcription FactorBasal transcription factor genesBenignBinswanger DiseaseBinswanger EncephalopathyBioassayBiologic ModelsBiological AssayBiological ModelsBiotechBiotechnologyBloodBlood Reticuloendothelial SystemBlood VesselsBlood monocyteBone marrow failureBrain InfarctionBrain Vascular AccidentCD8 CellCD8 T cellsCD8 lymphocyteCD8+ T cellCD8+ T-LymphocyteCD8-Positive LymphocytesCD8-Positive T-LymphocytesCNS Nervous SystemCRISPR approachCRISPR based approachCRISPR correctionCRISPR methodCRISPR methodologyCRISPR techniqueCRISPR technologyCRISPR toolsCRISPR-CAS-9CRISPR-based correctionCRISPR-based methodCRISPR-based techniqueCRISPR-based technologyCRISPR-based toolCRISPR/CAS approachCRISPR/Cas methodCRISPR/Cas technologyCRISPR/Cas9CRISPR/Cas9 technologyCachecticCachexiaCalcifiedCardiac Muscle CellsCardiac MyocytesCardiocyteCardiovascular DiseasesCas nuclease technologyCas9-based correctionCas9-mediated correctionCausalityCell BodyCell LineCell LineageCell modelCellLineCellsCellular modelCentral Nervous SystemCerebral StrokeCerebrovascular ApoplexyCerebrovascular StrokeCerebrumCharacteristicsChemicalsChronicChronic Progressive Subcortical EncephalopathyChronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperatureCicatrixClinicalClustered 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 technologyCo-cultureCocultivationCocultureCoculture TechniquesCollaborationsComplexCore FacilityCutaneousDNA mutationDeath RateDegos diseaseDental PorcelainDevelopmentDiagnosisDigestive TractDiseaseDisorderDrug TargetingDrug TherapyDrugsDysembryomaDysfunctionEndothelial CellsEndotheliumEtiologyExhibitsExpression SignatureEyeEyeballFibrosisFunctional disorderGI TractGI perforationGastrointestinal TractGastrointestinal tract structureGene AlterationGene Expression ProfileGene MutationGeneral Transcription Factor GeneGeneral Transcription FactorsGenesGeneticGenetic ChangeGenetic defectGenetic mutationGenetic studyGerm LayersHeartHeart Muscle CellsHeart failureHeart myocyteHematopoieticHumanHyper-IgE SyndromeHyperimmunoglobulin E SyndromeHyperimmunoglobulin E-Recurrent Infection SyndromeIL6-response factorImmuneImmunesIn VitroInflammationInflammatoryJob's SyndromeKinasesLIF-response factorLeiomyocyteLesionLiver FibrosisLungLung InflammationLung Respiratory SystemLung Tissue FibrosisMacrophageMalignantMalignant - descriptorMarrow monocyteMedicationMesenchymalMesenchymal Progenitor CellMesenchymal Stem CellsMesenchymal progenitorMesenchymal stromal/stem cellsMesodermMiceMice MammalsModel SystemModelingModern ManMolecularMonolayer cultureMonosomy XMorgagni-Turner syndromeMorgagni-Turner-Albright syndromeMurineMusMutationMyelogenousMyeloidMφNHLBINational Heart, Lung, and Blood InstituteNeuraxisOrganOrganoidsPBMCPathogenicityPathway interactionsPatientsPeripheral Blood Mononuclear CellPharmaceutical PreparationsPharmacological TreatmentPharmacotherapyPhenotypePhosphotransferase GenePhosphotransferasesPhysiologicPhysiologicalPhysiopathologyPlayPneumonitisPopulationPorcelainProductionPublicationsPulmonary FibrosisPulmonary InflammationResearchRoleSTAT3 loss of functionSTING1 geneSamplingScarsScienceScientific PublicationSepsisShereshevskii-Turner syndromeSignal PathwaySignal Transducer and Activator of Transcription 3SkinSmooth Muscle CellsSmooth Muscle MyocytesSmooth Muscle Tissue CellSomatic CellSpinalStat3 proteinStem Cell ResearchStimulator of Interferon GenesStrains Cell LinesStress Response SignalingStrokeSubcortical Arteriosclerotic EncephalopathySubcortical InfarctionsSubcortical InfarctsSubcortical LeukoencephalopathySyndromeSystemT8 CellsT8 LymphocytesTMEM173TechnologyTeratoid TumorTeratomaTestingTherapeuticToxicologyTranscription Factor Proto-OncogeneTranscription factor genesTransgenic MiceTransphosphorylasesTurner's SyndromeTurner-Albright syndromeUllrich-Turner syndromeUpregulationVascular DiseasesVascular DisorderVascular Smooth MuscleVascular calcificationVascularizationVasculitisXO syndromeadenosine deaminaseadenosine deaminase deficiencyadvanced diseaseadvanced illnessalimentary tractautoinflammatoryautosomebiobankbiological adaptation to stressbiorepositoryblood vessel disorderblood vessel occlusionbrain attackbrain infarctbrain vascularizationcGAMP STINGcGAMP-STINGcGAMP/STINGcGAS/STINGcalcificationcardiac failurecardiomyocytecardiovascular disordercausationcell replacement therapycell replacement treatmentcell typecerebralcerebral vascular accidentcerebral vascularizationcerebrovascular accidentchromosome XO syndromecultured cell linecyclic GMP-AMP synthase/STINGcytokinedental ceramicdevelopmentaldifferentiation protocoldigestive canaldisease causationdisease modeldisease phenotypedisorder modeldrug interventiondrug treatmentdrug/agentearly onsetexperienceexperimentexperimental researchexperimental studyexperimentsfibrosis in the lungfibrotic livergastrointestinal perforationgene correctedgene correctiongene defectgene editing platformgene editing systemgene editing technologygene editing toolsgene expression patterngene expression signaturegene-editing toolkitgenital dwarfismgenome mutationgenomic correctionhealthy volunteerhemopoietichepatic fibrosishiPSChuman iPShuman iPSChuman induced pluripotent cellhuman induced pluripotent stem cellshuman inducible pluripotent stem cellshuman inducible stem cellshypoimmunityiPSiPSCiPSC technologyiPSCsimmune deficiencyimmunodeficiencyimprovedin vivoinduced human pluripotent stem cellsinduced pluripotent cellinduced pluripotent stem cellinduced pluripotent stem cell technologyinduced pluripotent stem cells derived from patientsinduced pluripotent stem cells from patientsinducible pluripotent cellinducible pluripotent stem cellinfancyinfantileloss of function mutationlung failurelung fibrosismesenchymal stromal cellmesenchymal stromal progenitor cellsmesenchymal-derived stem cellsmodel of animalmonocytemonosomy X syndromemortality ratemortality ratiomutant allelenew therapeutic approachnew therapeutic interventionnew therapeutic strategiesnew therapy approachesnew treatment approachnew treatment strategynovelnovel therapeutic approachnovel therapeutic interventionnovel therapeutic strategiesnovel therapy approachovarian dwarfismovarian short stature syndromepathophysiologypathwaypatient 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 cellspharmaceutical interventionpharmacologicpharmacological interventionpharmacological therapypharmacology interventionpharmacology treatmentpharmacotherapeuticsporcelain Dental materials and fillingspseudonuchal infantilismpulmonarypulmonary failurerare conditionrare genetic diseaserare genetic disorderrare syndromereaction; crisisscRNA sequencingscRNA-seqself-renewself-renewalsingle cell RNA-seqsingle cell RNAseqsingle cell expression profilingsingle cell transcriptomic profilingsingle-cell RNA sequencingsmall moleculesocial rolestem cell studystress responsestress; reactionstrokedstrokessystemic inflammationsystemic inflammatory responsetherapeutic targettranscription factortranscriptional profiletranscriptional signaturevascularvascular dysfunctionvasculitidesvasculopathy
Description preview
The development and application of iPSC technology promises to revolutionize our understanding of disease mechanisms and improve the availability of treatment options. The ability to generate patient specific iPSC allows for the development of cell-based disease modeling where it has previously been extremely difficult to obtain sufficient amounts…
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