grant
AAV9fyhr gene replacement therapy for PDHA1 deficiency in the E75A mouse model
Organization UT SOUTHWESTERN MEDICAL CENTERLocation DALLAS, UNITED STATESPosted 1 May 2026Deadline 30 Apr 2028
NIHUS FederalResearch GrantFY2026Adeno-Associated VirusesAdverse effectsAffectAgeAge MonthsAnimal ModelAnimal Models and Related StudiesAnimalsArchivesAttentionAutopsyBiochemicalBirthBlindedBlood PlasmaBlood SerumBoard CertificationBody TissuesBody WeightBrain imagingCNS DiseasesCNS Nervous SystemCNS disorderCell Communication and SignalingCell SignalingCentral Nervous SystemCentral Nervous System DiseasesCentral Nervous System DisordersCerebrospinal FluidCessation of lifeClinicClinicalClinical ChemistryClinical TrialsCodonCodon NucleotidesCorpus CallosumCorpus CallosumsDNA TherapyDNA mutationDataDeathDependoparvovirusDependovirusDiagnosisDiseaseDisorderDorsal Root GangliaDoseEuthanasiaEvaluationExhibitsFutureGene DeliveryGene Expression MonitoringGene Expression Pattern AnalysisGene Expression ProfilingGene TransferGene Transfer ClinicalGenesGenetic ChangeGenetic InterventionGenetic defectGenetic mutationGenotypeGoalsHarvestHealthHepatotoxic effectHepatotoxicityHistopathologyHumanImmune responseImpairmentIncidenceIndividualInjectionsIntracellular Communication and SignalingIntrathecal InjectionsLaboratoriesLifeLinkLive BirthLiver ToxicityLongitudinal StudiesLongitudinal SurveysMediatingMedical centerMercy KillingMetabolicMetabolic acidosisMiceMice MammalsMitochondrial DiseasesMitochondrial DisordersModern ManMonitorMurineMusMutationNerve CellsNerve UnitNervous System DiseasesNervous System DisorderNeural CellNeuraxisNeurocyteNeurodevelopmental DisorderNeurologicNeurologic DisordersNeurologicalNeurological Development DisorderNeurological DisordersNeuromuscular DiseasesNeuronsNorth AmericaOrganPDHC Deficiency DiseaseParturitionPathologistPatientsPlasmaPlasma SerumPolyadenylationProductionPrognosisPyruvatePyruvate Dehydrogenase Complex Deficiency DiseaseRNA PolyadenylationReticuloendothelial System, Serum, PlasmaRiskSafetySerumSignal TransductionSignal Transduction SystemsSignalingSpinal GangliaStructural defectStructural malformationTestingTherapeuticTissuesToxic effectToxic effect on liver cellsToxicitiesToxicologyTranscript Expression AnalysesTranscript Expression AnalysisTransgenesTranslatingTranslational ResearchTranslational ScienceTranslationsTreatment EfficacyVariantVariationWild Type MouseWorkadeno associated virus groupagesanalyze gene expressionbehavior testbehavioral testbiological signal transductionbovine growth hormonebovine somatotropinbrain atrophybrain visualizationcerebral atrophycerebral spinal fluidclinical translationclinically translatablecortical atrophycurative interventioncurative therapeuticcurative therapycurative treatmentsdesigndesigningdetermine efficacydisease phenotypedorsal root ganglionefficacy analysisefficacy assessmentefficacy determinationefficacy evaluationefficacy examinationevaluate efficacyexamine efficacyexperiencefemale patientsfirst in manfirst-in-humangene expression analysisgene expression assaygene repair therapygene replacement therapygene therapygene-based therapygenetic therapygenome mutationgenomic therapyhepatic toxicityhepatoxicityhistopathologic examinationhistopathological examinationhost responseimmune system responseimmunoresponseintervention efficacylong-term studylongitudinal outcome studieslongitudinal research studymodel of animalmouse modelmurine modelmyoneural disordernecropsyneurodevelopmental diseaseneurological diseaseneuromuscular degenerative disorderneuromuscular disorderneuronalnovelold agepatients being femalepatients being womenpatients who are femalepostmortempostnatalpre-clinicalpreclinicalprogramspromoterpromotorprotein functionpyruvate dehydrogenase complex (PDHC) deficiencypyruvate dehydrogenase complex deficiencysafety assessmentsexsomidobovespinal fluidstructural abnormalitiesstructural anomaliestherapeutic efficacytherapy efficacytranscriptional profilingtransgenetransgene expressiontranslationtranslation researchtranslational investigationvectorvector biodistributionwildtype mousewomen patientsyounger age
Description preview
PROJECT SUMMARY/ABSTRACT
Primary Pyruvate Dehydrogenase Complex Deficiency (PDCD) is a rare but severe mitochondrial disorder,
with an estimated incidence of 1 in 50,000 to 75,000 live births in North America. The majority of cases are
caused by mutations in the X-linked PDHA1 gene. There is currently no curative therapy for PDHA1 deficiency,
and…
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