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Dystrophin Replacement
Organization UNIVERSITY OF WASHINGTONLocation SEATTLE, UNITED STATESPosted 1 Apr 1991Deadline 30 Apr 2026 ⚠️
NIHUS FederalResearch GrantFY20250-11 years old21+ years oldAAV deliveredAAV deliveryAAV vectorAAV-based deliveryAAV-based vectorAAV-based viral deliveryAAV-mediated deliveryAbnormal CellAddressAdeno-associated-virus-based deliveryAdipocytesAdipose CellAdolescentAdolescent YouthAdultAdult HumanAffectAgingAnimal ModelAnimal Models and Related StudiesAnimalsAreaBindingBiologic SciencesBiological SciencesBioscienceBiotechBiotechnologyBody TissuesCanine SpeciesCanis familiarisCapsidChildChild YouthChildren (0-21)Clinical TrialsDNA TherapyDataDevelopmentDiseaseDisorderDogsDogs MammalsDoseDucheneDuchenneDuchenne muscular dystrophyDuchenne-Griesinger syndromeDysfunctionDystrophinEligibilityEligibility DeterminationEllis-van Creveld (EvC) syndromeEncapsulatedEnhancer ElementsFat CellsFibrosisFunctional disorderFutureGene DeliveryGene TargetingGene Transfer ClinicalGene therapy trialGenesGenetic DiseasesGenetic Enhancer ElementGenetic InterventionGrantHumanHuman GeneticsImmunochemical ImmunologicImmunologicImmunologicalImmunologicallyImmunologicsInfusionInfusion proceduresInvestigationLibrariesLife SciencesLigandsLipocytesLiverMaintenanceMature LipocyteMature fat cellMediatingMessenger RNAMethodsMiceMice MammalsModern ManModificationMolecular InteractionMonitorMorphologyMurineMusMuscleMuscle CellsMuscle DiseaseMuscle DisordersMuscle FibersMuscle TissueMuscle satellite cellMuscular DiseasesMuscular DystrophiesMyocytesMyodystrophicaMyodystrophyMyopathic ConditionsMyopathic Diseases and SyndromesMyopathic disease or syndromeMyopathyMyotubesNamesNatural regenerationNeuromuscular DiseasesNucleocapsidOutcomePathologyPeptidesPhenotypePhysiopathologyProductionProgenitor CellsProtocol ScreeningPseudohypertrophic Muscular DystrophyRegenerationRhabdomyocyteSafetySerotypingSkeletal FiberSkeletal MuscleSkeletal Muscle CellSkeletal Muscle FiberSkeletal MyocytesSolidStriated MusclesStructureSurfaceSystemTestingTimeTissuesTransgenic OrganismsTreatment EfficacyVoluntary MuscleX-linked dilated cardiomyopathyX-linked muscular dystrophyX-linked recessive muscular dystrophyadeno-associated viral vectoradeno-associated viral vector deliveryadeno-associated virus deliveryadeno-associated virus mediated deliveryadeno-associated virus vectoradenovirus mediated deliveryadult animaladulthoodaged animalaged animalsaged miceaged mouseanimal old agebenign X-linked recessive muscular dystrophyboyscaninecell transductioncellular transductionchildhood pseudohypertrophic muscular dystrophyclassic X-linked recessive muscular dystrophycostdelivered with AAVdelivery vectordelivery vehicledelivery with AAVdesigndesigningdevelopmentaldomestic dogelderly animalelderly miceenhancer sequencegene repair therapygene therapygene transfer trialgene-based therapygenetic conditiongenetic disordergenetic enhancer sequencegenetic therapygenomic therapyhepatic body systemhepatic organ systemimprovedinfusionsintervention efficacyjuvenilejuvenile humankidsmRNAmature animalmicro-dystrophinmicrodystrophinmild X-linked recessive muscular dystrophyminiaturizeminiaturizedmodel of animalmuscle dystrophymuscle regenerationmuscularmuscular disordermyoneural disordernamenamednamingneuromuscular degenerative disorderneuromuscular disordernovelold animalsold miceparticlepathophysiologypre-clinical studypreclinical studypreventpreventingprogressive muscular dystrophy of childhoodpromoterpromotorpseudohypertrophic adult muscular dystrophypseudohypertrophic muscular paralysisregeneratesatellite cellscale upscreeningscreeningsself assemblystem cellstech developmenttechnology developmenttherapeutic efficacytherapy efficacytransduced cellstransgenicvectoryoung manyoung menyoungster
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Project Summary
Gene therapy for Duchenne muscular dystrophy (DMD) is a promising method to treat this devastating
disorder, and 3 companies have recently initiated human clinical trials of AAV/micro-dystrophin infusion. This
approach has shown significant potential in pre-clinical studies, and early data from the trials are encouraging.
It is…
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