grant
Genomically rewritten and tailored humanized mouse models for various organ disorders
Organization NEW YORK UNIVERSITY SCHOOL OF MEDICINELocation NEW YORK, UNITED STATESPosted 1 May 2025Deadline 28 Feb 2029
NIHUS FederalResearch GrantFY2025AD dementiaANK1ANK1 geneAdvisory CommitteesAffectAge related macular degenerationAge-Related MaculopathyAllelesAllelomorphsAlternate SplicingAlternative RNA SplicingAlternative SplicingAlzheimer Type DementiaAlzheimer disease dementiaAlzheimer sclerosisAlzheimer syndromeAlzheimer'sAlzheimer's DiseaseAlzheimers DementiaAnatomic SitesAnatomic structuresAnatomyAnimal ModelAnimal Models and Related StudiesAnimalsAniridiaAnkyrin 1AtlasesAutoimmune StatusAutoimmunityAwardBehaviorBipolar Affective PsychosisBipolar DisorderBody SystemBody TissuesBrainBrain Nervous SystemBreedingCRISPR based therapeuticsCRISPR based treatmentCRISPR therapeuticsCRISPR treatmentCRISPR-Cas based therapeuticsCRISPR-based disease therapeuticsCRISPR-based therapyCRISPR/Cas therapeuticsCRISPR/Cas9 therapeuticsCRISPR/Cas9 therapyCRISPR/Cas9 treatmentCRISPR/Cas9-based therapyCardiovascularCardiovascular Body SystemCardiovascular DiseasesCardiovascular Organ SystemCardiovascular systemCas9 based therapeuticsCell BodyCell NucleusCellsClone CellsClustered Regularly Interspaced Short Palindromic Repeats based therapeuticsClustered Regularly Interspaced Short Palindromic Repeats therapeuticsCommunitiesComplementComplement ProteinsCoronary ArteriosclerosisCoronary Artery DiseaseCoronary Artery DisorderCoronary AtherosclerosisDNADNA SequenceDNA TherapyDNA deliveryDeoxyribonucleic AcidDepositDepositionDiabetes MellitusDiathesisDiseaseDisease susceptibilityDisorderDominantly-Inherited Spinocerebellar AtaxiasEncephalonEyeEyeballFunctional RNAFundingGEM modelGEMM modelGWA studyGWASGene ClusterGene ExpressionGene Transfer ClinicalGenesGenetic DiseasesGenetic InterventionGenetically Engineered MouseGenomeGenomicsGrantHandHeart VascularHumanHuman PathologyIFNImmuneImmune DiseasesImmune DisordersImmune DysfunctionImmune System DiseasesImmune System DisorderImmune System DysfunctionImmune System and Related DisordersImmune systemImmunesImmunologic DiseasesImmunological DiseasesImmunological DysfunctionImmunological System DysfunctionInstructionInterferonsInternationalIntervening SequencesIntronsInvestigatorsKnowledgeLocationManic-Depressive PsychosisManualsMetabolic dysfunctionMethodsMiceMice MammalsModelingModern ManMouse ES CellMouse ESCMouse Embryonic ProgenitorMouse Embryonic Stem CellsMurineMusMuscleMuscle TissueNHGRINHLBINIAIDNIDDKNIGMSNIMHNINDSNational Center for Human Genome ResearchNational Heart, Lung, and Blood InstituteNational Human Genome Research InstituteNational Institute of Allergy and Infectious DiseaseNational Institute of Diabetes and Digestive and Kidney DiseasesNational Institute of General Medical SciencesNational Institute of Mental HealthNational Institute of Neurological Diseases and StrokeNational Institute of Neurological Disorders and StrokeNational Institutes of HealthNatureNeurophysiology - biologic functionNoncoding RNANontranslated RNANucleusOn-Line SystemsOnline SystemsOrganOrgan SystemPancreasPancreaticPatternPhenotypePrimary Senile Degenerative DementiaProcessProductionR24RNA SeqRNA sequencingRNAseqReagentResearch PersonnelResearch ResourcesResearchersResourcesRetinaRisk-associated variantSchizophreniaSchizophrenic DisordersSpinocerebellar AtaxiasSpinocerebellar AtrophiesStructureTask ForcesTechniquesTechnologyTestingTherapeutic InterventionTissuesTranslatingTrinucleotide RepeatsTriplet RepeatsUnited States National Institutes of HealthUntranslated RNAVascular EndotheliumViralWorkWritingadvisory teamage dependent macular degenerationage induced macular degenerationage related macular diseaseage related macular dystrophyatherosclerotic coronary diseasebiomarker arraybiomarker panelbipolar affective disorderbipolar diseasebipolar illnessbipolar mood disordercardiovascular disordercell typecirculatory systemclinical relevanceclinically relevantcodon reiterationcomplementationcoronary arterial diseasedark matterdata portaldata sharing portaldeliver DNAdementia praecoxdiabetesdisease phenotypegene repair therapygene therapygene-based therapygenetic conditiongenetic disordergenetic therapygenetically engineered mouse modelgenetically engineered murine modelgenome sciencesgenome wide associationgenome wide association scangenome wide association studygenomewide association scangenomewide association studygenomic sciencegenomic therapyhandshuman DNAhuman diseasehuman modelhumanized micehumanized mouseimprovedinterestintervention therapyliability to diseasemESCmanic depressive disordermanic depressive illnessmarker panelmodel of animalmodel of humanmouse genomemouse modelmurine ES cellsmurine ESCmurine embryonic progenitormurine embryonic stem cellmurine modelmuscularneuralneural functionneuropsychiatric diseaseneuropsychiatric disordernoncodingoligonucleotide drugoligonucleotide therapeuticsoligonucleotide therapiesoligonucleotide treatmentonline computerprimary degenerative dementiaprospectiveprotective alleleprotective variantresponserisk allelerisk generisk genotyperisk locirisk locusrisk variantscRNA sequencingscRNA-seqschizophrenicsenile dementia of the Alzheimer typesenile macular diseasesingle cell RNA-seqsingle cell RNAseqsingle cell expression profilingsingle cell transcriptomic profilingsingle-cell RNA sequencingspatial and temporalspatial temporalspatiotemporaltherapeutic evaluationtherapeutic testingtranscriptome sequencingtranscriptomic sequencingweb basedwhole genome association analysiswhole genome association study
Description preview
Summary
This proposal leverages Big DNA technology to produce mouse models of a variety of human diseases. The
underlying hypothesis tested in our earlier work and continued in this proposal is that changes in human non-
coding DNA that underlie human disease susceptibility will translate into appropriate disease phenotypes in
the mouse when the…
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