grant
Leveraging modulation of polyamine metabolism for therapeutic advantage in genetic disorders
Organization MICHIGAN STATE UNIVERSITYLocation EAST LANSING, UNITED STATESPosted 1 Feb 2023Deadline 31 Jan 2028
NIHUS FederalResearch GrantFY20250-4 weeks old1,4-Butanediamine1,4-Diaminobutane21+ years oldAdultAdult HumanAffectAfter CareAfter-TreatmentAftercareAnimal ModelAnimal Models and Related StudiesAnimalsAssayBehavioralBioassayBiochemicalBiochemical PathwayBiochemistryBiological AssayBiological ChemistryBiologyBlood PlasmaBlood SampleBlood specimenBody TissuesC-terminalCRISPR approachCRISPR based approachCRISPR methodCRISPR methodologyCRISPR techniqueCRISPR technologyCRISPR toolsCRISPR-CAS-9CRISPR-based methodCRISPR-based techniqueCRISPR-based technologyCRISPR-based toolCRISPR/CAS approachCRISPR/Cas methodCRISPR/Cas technologyCRISPR/Cas9CRISPR/Cas9 technologyCas nuclease technologyCell BodyCell LineCellLineCellsClinicalClinical DataClustered 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 technologyDFMODL-alpha-DifluoromethylornithineDL-α-DifluoromethylornithineDNA mutationDataData AnalysesData AnalysisDiagnosisDiseaseDisorderDominant Genetic ConditionsDominant traitDoseDrug KineticsDrug TherapyDrugsEffectivenessEflornithineEnzyme GeneEnzymesFDA approvedFamilyGene AlterationGene MutationGene variantGenesGeneticGenetic ChangeGenetic DiseasesGenetic DominantGenetic defectGenetic mutationGoalsHairHeterozygoteHistologyHumanHuman GeneticsIF-5AIn VitroIndividualIntermediary MetabolismL-Ornithine carboxy-lyaseLeadLightLinkMedicationMetabolicMetabolic NetworksMetabolic PathwayMetabolic ProcessesMetabolismMiceMice MammalsModelingModern ManMolecularMonitorMurineMusMutationNeurodevelopmental DisorderNeurological Development DisorderNeurologyNewborn InfantNewbornsOrnithine Carboxy-lyaseOrnithine DecarboxylaseOrphan DiseasePathogenicityPathologicPathologyPathway interactionsPatientsPb elementPharmaceutical PreparationsPharmacokineticsPharmacological TreatmentPharmacotherapyPhenotypePhotoradiationPlasmaPlasma SerumPolyamine CompoundPolyaminesPublishingPutrescineRare DiseasesRare DisorderRegulationReticuloendothelial System, Serum, PlasmaSamplingSentinelSkinSnyder-Robinson Intellectual Disability SyndromeSnyder-Robinson syndromeSpermidineSpermidine AminopropyltransferaseSpermineSpermine SynthaseSpermine SynthetaseStrains Cell LinesSymptomsSyndromeTestingTetramethylenediamineThe Jackson LaboratoryTherapeuticTissue SampleTissuesTransgenic OrganismsTreatment EffectivenessVariantVariationWhole BloodX-linked mental retardation-thin habitus-osteoporosis-kyphoscoliosis syndromeadulthoodallelic variantalleviate symptomalpha-Difluoromethyl Ornithinealpha-Difluoromethylornithineameliorating symptomanalogautosomebehavior responsebehavioral responseclinical carecultured cell linedata interpretationde novo mutationde novo variantdecrease symptomdesigndesigningdifferential expressiondifferentially expresseddifluoromethylornithinedisease modeldisorder modeldrug interventiondrug treatmentdrug/agenteIF-4DeIF-5AeIF5A proteineffectiveness testingeukaryotic initiation factor-4Dfewer symptomsgain of functiongain of function mutationgene defectgene testinggene-based testinggenetic conditiongenetic disordergenetic testinggenetic variantgenome editinggenome mutationgenomic editinggenomic variantglobal developmental delayglobal gene expressionglobal transcription profileheavy metal Pbheavy metal leadheterozygosityimprovedin vivoinhibitorloss of functionloss of function mutationlymphoblastoid cell linemetabolism measurementmetabolomicsmetabonomicsmodel of animalmouse modelmurine modelmutant alleleneurodevelopmental diseasenew drug treatmentsnew drugsnew pharmacological therapeuticnew therapeuticsnew therapynewborn childnewborn childrennext generation therapeuticsnovelnovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel therapeuticsnovel therapyorphan disorderpathwaypharmaceutical interventionpharmacologicpharmacological interventionpharmacological therapypharmacology interventionpharmacology treatmentpharmacotherapeuticspost treatmentreduce symptomsrelieves symptomsresponseresponse to therapyresponse to treatmentscreeningscreeningssymptom alleviationsymptom reductionsymptom relieftargeted agenttherapeutic responsetherapy responsetooltranscriptional differencestranscriptometranscriptomicstransgenictreatment responsetreatment responsiveness
Description preview
ABSTRACT
We are at the forefront of investigating novel neurodevelopmental disorders associated with polyamines. Our
overarching hypothesis is that mutations in genes of the polyamine pathway result in pathologically unbalanced
polyamine profiles in affected individuals that lead to neurodevelopmental disorders. Specifically, Bachmann-
Bupp…
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