grant

Harnessing mitochondria transfer pathways to ameliorate Leigh Syndrome-like disease

Organization WASHINGTON UNIVERSITYLocation SAINT LOUIS, UNITED STATESPosted 1 Jul 2024Deadline 30 Jun 2029
NIHUS FederalResearch GrantFY2025AffectAutoregulationB220BiodistributionBiologicalBody TissuesBone MarrowBone Marrow Cell TransplantationBone Marrow GraftingBone Marrow Reticuloendothelial SystemBone Marrow TransplantBone Marrow TransplantationBrainBrain Nervous SystemCD45Cell BodyCellsCellular Metabolic ProcessCessation of lifeChildhoodComplexDataDeathDefectDiseaseDisorderDyskinesia SyndromesEncephalonEnergy ExpenditureEnergy MetabolismEngraftmentGP180Genetic PolymorphismGliaGlial CellsHematopoieticHereditaryHomeostasisImmuneImmunesIn VitroInfant MortalityInfant Mortality TotalInflammationInheritedIntercellular nanotubesIntermediary MetabolismInterventionKolliker's reticulumLY5LaboratoriesLeigh DiseaseLeigh SyndromeMacrophageMarrow TransplantationMediatingMetabolicMetabolic ProcessesMetabolismMiceMice MammalsMitochondriaMitochondrial DNAMitochondrial DiseasesMitochondrial DisordersModelingMorbidityMorbidity - disease rateMovement Disorder SyndromesMovement DisordersMurineMusNatural regenerationNerve CellsNerve DegenerationNerve UnitNervous System PhysiologyNeural CellNeurocyteNeurogliaNeuroglial CellsNeurologicNeurologic ManifestationsNeurologic Signs and SymptomsNeurologic SymptomsNeurologic functionNeurologicalNeurological ManifestationsNeurological Signs and SymptomsNeurological functionNeuron DegenerationNeuronsNon-neuronal cellNonneuronal cellOrganPTPRCPTPRC genePathway interactionsPatientsPhysiological HomeostasisProcessRegenerationReporterReportingResearchSeveritiesSeverity of illnessSourceSubacute Necrotizing EncephalomyelitisSubacute Necrotizing EncephalomyelopathySubacute Necrotizing EncephalopathyT200TechniquesTechnologyTherapeuticTissuesTransplantationWorkbiologiccell metabolismcell typecellular metabaolismdeath among infantsdeath in first year of lifedeath in infancydeath in infantsdisease severityearly childhoodeffective therapyeffective treatmentenzyme activityextracellular vesicleshematopoietic engraftmenthemopoieticimprovedin vivoinfant deathinfant demiseinfantile deathinnovateinnovationinnovativelife spanlifespanmembrane nanotubesmembranous nanotubesmitochondrialmitochondrial metabolismmortalitymortality in infantsmtDNAnerve cementnervous system functionneural degenerationneural manifestationneurodegenerationneurodegenerativeneurological degenerationneuronalneuronal degenerationnew drug treatmentsnew drugsnew pharmacological therapeuticnew therapeutic approachnew therapeutic interventionnew therapeutic strategiesnew therapeuticsnew therapynew therapy approachesnew treatment approachnew treatment strategynext generation therapeuticsnovelnovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel therapeutic approachnovel therapeutic interventionnovel therapeutic strategiesnovel therapeuticsnovel therapynovel therapy approachpathwaypediatricpharmacologicpolymorphismpostnatalregeneraterespiratoryself-renewself-renewaltargeted drug therapytargeted drug treatmentstargeted therapeutictargeted therapeutic agentstargeted therapytargeted treatmenttransplanttunneling nanotubes
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PROJECT SUMMARY
Leigh Syndrome (LS) is an inherited mitochondrial disease that presents with prominent neurologic symptoms

and death in childhood. There are currently no effective therapies for this devastating disease, highlighting an

urgent need to identify novel biological pathways that can be targeted therapeutically to treat LS and other…

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Harnessing mitochondria transfer pathways to ameliorate Leigh Syndrome-like disease — WASHINGTON UNIVERSITY | UNITED STA | Dev Procure