grant
Prenatal Treatment of Down Syndrome to Improve Brain Development and Neurocognition
Organization NATIONAL HUMAN GENOME RESEARCH INSTITUTELocation UNITED STATES
NIHUS FederalResearch GrantFY2025AdoptionAffectAgeAmniocentesisAmyloid A4 Protein PrecursorAmyloid Protein PrecursorAmyloid beta-Protein PrecursorAmyloid β-Protein PrecursorAntiinflammatory EffectAntioxidantsAssayBioassayBiologic ModelsBiological AssayBiological ModelsBody SystemBrainBrain Nervous SystemCardiovascularCardiovascular Body SystemCardiovascular Organ SystemCardiovascular systemCell AgingCell Communication and SignalingCell LineCell SenescenceCell SignalingCellLineCells Placenta-TissueCellular AgingCellular ExpansionCellular GrowthCellular SenescenceCharacteristicsChileChorionic Villi BiopsyChorionic Villi SamplingChromosome 16Chromosome 21ClinicalClinical ManagementCollaborationsCollectionCytogeneticsDataData BasesData SetDatabasesDeveloping fetusDevelopmentDiagnosisDiagnostic MethodDiagnostic ProcedureDiagnostic TechniqueDiseaseDisorderDown SyndromeDrugsEarly treatmentEmbryoEmbryonicEncephalonEvaluationExhibitsExpression SignatureFetal DevelopmentFetal TherapiesFetusFibroblastsFutureGene ExpressionGene Expression MonitoringGene Expression Pattern AnalysisGene Expression ProfileGene Expression ProfilingGene ProteinsGeneralized GrowthGenesGenomicsGoalsGrowthHeart VascularHortega cellHumanHuman ChromosomesIFNIndividualInfantIntellectual disabilityIntellectual functioning disabilityIntellectual limitationInterferonsIntracellular Communication and SignalingLaboratoriesLangdon Down syndromeLibrariesLifeLiteratureMacrophageMapsMeasuresMedical centerMedicationMiceMice MammalsMicrogliaMitochondriaModel SystemModelingModern ManMolecularMongolismMurineMusMusculoskeletal SystemMφNational Institute of AgingNational Institute on AgingNetwork-basedNeural Stem CellNeurocognitionNeurologicNeurologicalNormal PlacentomaOrgan SystemOutcomeOxidative StressPathway interactionsPharmaceutical PreparationsPhenotypePlacentaPlacenta Embryonic TissuePlacental DevelopmentPlacentationPlacentomePregnant WomenProliferatingProtein Gene ProductsProteomePublishingQOL improvementRNA SeqRNA sequencingRNAseqReplicative SenescenceResearchRoleSOD-1SOD-1 proteinSOD1SOD1 geneSOD1 gene productSafetySamplingSignal TransductionSignal Transduction SystemsSignalingStrains Cell LinesSupportive TherapySupportive careTherapeuticTherapeutic InterventionTimeTissue GrowthTranscript Expression AnalysesTranscript Expression AnalysisTrisomyTrisomy 16 DS mouseTrisomy 21Ts1CjeTs65DnWorkagesamyloid precursor proteinanalyze gene expressionantenatalantepartumanti-inflammatory effectbehavior phenotypebehavioral phenotypingbio-informatics toolbioinformatics toolbiological adaptation to stressbiological signal transductioncell growthchorionic villous samplingchromosome 21 trisomychromosome 21 trisomy syndromecirculatory systemcongenital acromicria syndromecultured cell linedata basedata diversitydevelopmentaldiverse datadrug candidatedrug/agentearly therapyexpectant motherexpectant womenexpecting motherexpecting womenfetalfetus therapygene expression analysisgene expression assaygene expression patterngene expression signaturegitter cellglobal gene expressionglobal transcription profileiPSiPSCiPSCsimprovedimprovements in QOLimprovements in quality of lifein utero therapyin vitro Modelindividuals who are pregnantinduced pluripotent cellinduced pluripotent stem cellinducible pluripotent cellinducible pluripotent stem cellinsightintellectual and developmental disabilityinter-individual variabilityinter-individual variationinterestintervention therapylife spanlifespanlimited intellectual functioninglocomotor systemmesogliamicroglial cellmicrogliocytemitochondrialmorbus Downmouse Trisomy 16mouse Ts1Cjemouse Ts65Dnmouse modelmurine modelnerve stem cellneural inflammationneural precursorneural precursor cellneural progenitorneural progenitor cellsneural stem and progenitor cellsneurogenic progenitorsneurogenic stem cellneuroinflammationneuroinflammatoryneuron progenitorsneuronal progenitorneuronal progenitor cellsneuronal stem cellsneuroprogenitornovelontogenypathwaypeople who are pregnantperivascular glial cellpostnatalpre-clinicalpreclinicalpregnant femalespregnant motherspregnant peoplepregnant populationsprenatalprenatal screeningprenatal testingprenatal therapyprogenitor and neural stem cellsprotein expressionpseudohypertrophic progressive muscular dystrophyquality of life improvementracial diversityracially diversereaction; crisisreplicative agingresponseresponse to therapyresponse to treatmentscreeningscreeningssenescencesenescentsexskillssmall moleculesocial rolestress responsestress; reactionsuperoxide dismutase 1therapeutic responsetherapy responsethose who are pregnanttranscriptional profiletranscriptional profilingtranscriptional signaturetranscriptometranscriptome sequencingtranscriptomic sequencingtranscriptomicstreatment responsetreatment responsivenesstrisomy 21 syndromeunbornwomen who are pregnant
Description preview
During the past year, we achieved the following progress towards our goals and objectives:
1) Molecular and cellular phenotyping of induced pluripotent stem cells (iPSCs) and iPSC-derived neural progenitor cells (NPCs) from individuals with T21 and age- and sex-matched euploid individuals.
We generated a novel, genetically diverse panel of age-…
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