grant
Investigating the Feasibility of Gene Therapy for the Treatment of TBRS
Organization WASHINGTON UNIVERSITYLocation SAINT LOUIS, UNITED STATESPosted 1 Mar 2026Deadline 28 Feb 2028
NIHUS FederalResearch GrantFY202621+ years oldAAV deliveredAAV deliveryAAV vectorAAV-based deliveryAAV-based vectorAAV-based viral deliveryAAV-mediated deliveryASDAddressAdeno-Associated VirusesAdeno-associated-virus-based deliveryAdultAdult HumanAffectAllelic LossAnimalsAutismAutistic DisorderBehaviorBehavioralBody TissuesBrainBrain Nervous SystemCRE RecombinaseCandidate Disease GeneCandidate GeneCell BodyCell DifferentiationCell Differentiation processCellsCerebral cortexCollaborationsComputational BiologyCytosineDNA MethylationDNA MethyltransferaseDNA Modification MethylasesDNA Modification MethyltransferasesDNA TherapyDNA mutationDNA-MethyltransferasesDecreased Muscle ToneDependoparvovirusDependovirusDepositDepositionDevelopmentDinucleoside PhosphatesDiseaseDisorderDnmtDoseEarly Infantile AutismEncephalonEnsureEnterobacteria phage P1 Cre recombinaseEnvironmentEpigeneticEpigenetic ChangeEpigenetic MechanismEpigenetic ProcessExhibitsFaceFamilyFosteringFoundationsGene ExpressionGene Expression MonitoringGene Expression Pattern AnalysisGene Expression ProfilingGene TargetingGene Transfer ClinicalGeneralized GrowthGenesGeneticGenetic ChangeGenetic DiseasesGenetic InterventionGenetic defectGenetic mutationGenomeGenomicsGoalsGrowthHead circumferenceHealthHeightHumanHypomyotoniaHypotoniaIndustry StandardInfantile AutismIntellectual disabilityIntellectual functioning disabilityIntellectual limitationInvestigatorsJoint HypermobilityJoint LaxityKanner's SyndromeLearning DisabilitiesLearning disabilityLifeLife ExpectancyLinkLocationLoss of HeterozygosityMediatingMentorshipMethodsMethylationMiceMice MammalsModern ManModification MethylasesMolecularMurineMusMuscle HypotonyMuscle Tone PoorMuscle hypotoniaMuscular HypotoniaMutateMutationNerve CellsNerve UnitNeural CellNeurocyteNeurodevelopmental DisorderNeurological Development DisorderNeuronal DifferentiationNeuronsObesityOrganismOutcomePatientsPersonsPhenotypePlayProtein MethylasesProtein MethyltransferasesQOLQuality of lifeRNA SeqRNA sequencingRNAseqRahman SyndromeResearchResearch PersonnelResearch ResourcesResearchersResolutionResourcesRoleScientific InquirySeizuresSingle-Nucleus SequencingSite-Specific DNA-methyltransferaseSpecific qualifier valueSpecifiedSymptomsTamoxifenTechnologyTestingTherapeuticTimeTissue GrowthTissuesTranscript Expression AnalysesTranscript Expression AnalysisUniversitiesWashingtonWorkadeno associated virus groupadeno-associated viral vectoradeno-associated viral vector deliveryadeno-associated virus deliveryadeno-associated virus mediated deliveryadeno-associated virus vectoradenovirus mediated deliveryadiposityadulthoodanalyze gene expressionautism spectral disorderautism spectrum disorderautistic spectrum disorderbacteriophage P1 recombinase Crecell typecellular differentiationcomputer biologycorpulencedelivered with AAVdelivery with AAVdevelopmentaldinucleotidedisease modeldisorder modeldosageepigeneticallyexperimentexperimental researchexperimental studyexperimentsfacesfacialfeasibility testingfunctional outcomesfunctional restorationgene expression analysisgene expression assaygene repair therapygene replacementgene replacement therapygene therapygene-based therapygene-based treatmentgene-directed therapygene-targeted therapygene-targeted treatmentgenetic conditiongenetic disordergenetic therapygenome mutationgenomic therapyimaging platformimprovedimproved outcomein vivoin vivo Modelinduced Creinducible Creinnovateinnovationinnovativeinsightintellectual and developmental disabilitylimited intellectual functioningliving systemmethylation patternmethylomicsmouse modelmurine modelneuralneurodevelopmental diseaseneuron developmentneuronalneuronal developmentontogenyoverexpressoverexpressionpost-natal periodpostnatalpostnatal periodprotein expressionresolutionsrestorationrestore functionrestore functionalityrestore lost functionsNuc-Seqsingle nucleus RNA-sequencingsingle nucleus seqsingle-nucleus RNA-seqsnRNA sequencingsnRNA-seqsocial rolespatial RNA sequencingspatial gene expression analysisspatial gene expression profilingspatial resolved transcriptome sequencingspatial transcriptome analysisspatial transcriptome profilingspatial transcriptome sequencingspatial transcriptomicsspatially resolved transcriptomicsspatio transcriptomicssynapse formationsynapse functionsynaptic functionsynaptic pruningsynaptogenesistargeted drug therapytargeted drug treatmentstargeted therapeutictargeted therapeutic agentstargeted therapytargeted treatmenttooltraittranscriptional profilingtranscriptome sequencingtranscriptomic sequencingtranscriptomicsvector
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PROJECT SUMMARY
Neurodevelopmental disorders (NDDs) often result from mutations in genes essential for brain
development and function. Recent advances in gene replacement therapy have shown promise for
rescuing molecular and behavioral deficits in mouse models, even when gene restoration occurs
postnatally. However, the context and feasibility of…
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