grant
Microglia replacement using engineered HSCs for treatment of leukodystrophies
Organization UNIVERSITY OF PENNSYLVANIALocation PHILADELPHIA, UNITED STATESPosted 15 Apr 2025Deadline 31 Mar 2030
NIHUS FederalResearch GrantFY2025AddressAdoptive TransferAllogenicAnimal ModelAnimal Models and Related StudiesAnimalsAutologousBM Stem CellBM derived progenitorBM progenitorBM- derived Stem CellsBiochemicalBiologyBody TissuesBone Marrow Stem CellBone Marrow progenitorBrainBrain DiseasesBrain DisordersBrain Nervous SystemBussulfamBusulfanBusulfanumCD115CD115 GeneCNS Nervous SystemCSF1RCSF1R geneCSFMRCell BodyCell CompartmentationCell CompartmentationsCellsCellular immunotherapyCentral Nervous SystemCessation of lifeChildhoodClinicClinicalColony Stimulating Factor 1 Receptor GeneD-Galactosyl-N-acylsphingosine galactohydrolaseDNA TherapyDNA mutationDataDeathDegenerative Neurologic DisordersDiffuse Globoid Body SclerosisDiseaseDisease ProgressionDisorderDoseDrugsEncephalonEncephalon DiseasesEngineered GeneEngineeringEngraftmentEnsureEnvironmentEnzyme GeneEnzymesFDA approvedFosteringGEM modelGEMM modelGalactocerebrosidaseGalactosylceramidase Deficiency DiseaseGalactosylceramide GalactosidaseGalactosylceramide beta-GalactosidaseGalactosylcerebroside beta-GalactosidaseGene ModifiedGene Transfer ClinicalGenetic ChangeGenetic InterventionGenetic defectGenetic mutationGenetically Engineered MouseGloboid LeukodystrophyGloboid cell leukodystrophyGvHDHSC differentiationHSC transplantationHealthHematopoiesisHematopoieticHematopoietic Cellular Control MechanismsHematopoietic Stem Cell TransplantHematopoietic Stem Cell TransplantationHomologous Wasting DiseaseHortega cellHumanHydrolaseHydrolase Family GeneHydrolase GeneImmune systemInfantInfectionInjectionsIntracranial CNS DisordersIntracranial Central Nervous System DisordersIntrathecal InjectionsKnowledgeKrabbe DiseaseKrabbe leukodystrophyLysosomal Enzyme DisordersLysosomal Storage DiseasesM mulattaM. mulattaMacaca mulattaMacaca rhesusMacrophageMediatingMedicationMethodsMiceMice MammalsMicrogliaModalityModelingModern ManModificationMolecularMorbidityMorbidity - disease rateMurineMusMutationMφNHP modelsNervous System Degenerative DiseasesNeural Degenerative DiseasesNeural degenerative DisordersNeuraxisNeurodegenerative DiseasesNeurodegenerative DisordersNeurologic Degenerative ConditionsPathologyPatientsPeripheralPharmaceutical PreparationsPhenotypePre-Clinical ModelPreclinical ModelsRegimenReportingResistanceRhesusRhesus MacaqueRhesus MonkeyRodentRodentiaRodents MammalsRoleRunt DiseaseSafetySulfabutinSymptomsTestingTherapeuticTimeTissuesToxic effectToxicitiesTranslatingTranslationsTransplantationTreatment EfficacyValidationVariantVariationViralWorkbeta galactocerebrosidase deficiencyblood cell formationblood stem cell transplantationbone marrow derived progenitorbone marrow derived stem cellsbone marrow stromal cellbone marrow stromal stem cellbrain cellc-FMSc-fms Genesc-fms Proto-Oncogenescell-based immunotherapyclinical translationclinically translatableconditioningcross reactivitydegenerative diseases of motor and sensory neuronsdegenerative neurological diseasesdesigndesigningdevelop therapydiffuse globoid cell cerebral sclerosisdisease modeldisorder modeldrug/agentexperiencegalactocerebrosidase (GALC) deficiencygalactocerebrosidase deficiencygalactosylceramidasegalactosylceramide beta-galactosidase deficiencygalactosylceramide deficiencygalactosylceramide lipidosisgalactosylsphingosine lipidosisgene modificationgene repair therapygene signaturesgene therapygene transplantationgene transplantation for gene therapygene-based therapygenetic signaturegenetic therapygenetically engineered mouse modelgenetically engineered murine modelgenetically modifiedgenome mutationgenomic therapygitter cellgloboid cell cerebral sclerosisgloboid cell sclerosisgraft versus host diseasegraft vs host diseasegraft vs. host diseasehematopoietic cell transplantationhematopoietic cellular transplantationhematopoietic progenitor cell differentiationhematopoietic progenitor cell transplantationhematopoietic progenitor differentiationhematopoietic stem cell differentiationhemopoietichuman subjectimmune cell therapyimprovedinborn lysosomal enzyme disorderinfancyinfantileinhibitorintervention developmentintervention efficacyleukodystrophylysosomal diseaselysosomal disorderlysosome storage diseasesmesogliamicroglial cellmicrogliocytemodel of animalmortalitymouse modelmurine modelneurodegenerative illnessnon-human primatenonhuman primatenonhuman primate modelsoverexpressoverexpressionpediatricperivascular glial cellpre-clinicalpreclinicalprime editingpsychosine lipidosisresistantrestorationsocial rolestandard of caresynergismtherapeutic efficacytherapy developmenttherapy efficacytranslationtranslational opportunitiestranslational potentialtransplanttransplant modeltreatment developmentvalidations
Description preview
Summary:
Globoid cell leukodystrophy (GLD, Krabbe) is a fatal pediatric neurodegenerative disease caused by mutation of
the lysosomal enzyme galactosylceramidase (GALC). The greatest hurdle to curing GLD is treatment of central
nervous system (CNS) pathology. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is standard of
care,…
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