grant
Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center - Seattle
Organization UNIVERSITY OF WASHINGTONLocation SEATTLE, UNITED STATESPosted 7 May 2014Deadline 31 Aug 2028
NIHUS FederalResearch GrantFY2025AAV deliveredAAV deliveryAAV-based deliveryAAV-based viral deliveryAAV-mediated deliveryAdeno-associated-virus-based deliveryAdvocacyAnimal ModelAnimal Models and Related StudiesAnimalsAntigenic DeterminantsBinding DeterminantsBiologicalCharacteristicsClinicalClinical ResearchClinical StudyClinical TreatmentClinical TrialsClinical Trials DesignCohort StudiesConcurrent StudiesDNA TherapyData AnalysesData AnalysisData SetDevelopmentDiseaseDisease ProgressionDisorderDoseDose LimitingDucheneDuchenneDuchenne muscular dystrophyDuchenne-Griesinger syndromeDystrophinEducationEducation and OutreachEducational ActivitiesEducational aspectsEllis-van Creveld (EvC) syndromeEnsureEpitopesFSHDFacioscapulohumeralFacioscapulohumeral AtrophyFacioscapulohumeral Muscular DystrophyFacioscapulohumeral Type Progressive Muscular DystrophyFamilyFasioscapulohumeral Muscular DystrophyFellowshipFoundationsFutureGene DeliveryGene Transfer ClinicalGenetic InterventionGoalsHumanHuman CharacteristicsHuman NatureInfrastructureInstruction and OutreachIntervening Protein SequenceInvestigatorsLandouzy Dejerine muscular dystrophyLandouzy-Dejerine DystrophyLeadMR ImagingMR TomographyMRIMRIsMagnetic Resonance ImagingMeasurementMeasuresMediatingMedical Imaging, Magnetic Resonance / Nuclear Magnetic ResonanceMentorsMethodsMiniature SwineMinipigsModern ManMolecularMonitorMuscular DystrophiesMyocardiumMyodystrophicaMyodystrophyNMR ImagingNMR TomographyNuclear Magnetic Resonance ImagingOn-Line SystemsOnline SystemsPatient advocacyPatientsPb elementPerformancePostdocPostdoctoral FellowPre-Clinical ModelPreclinical ModelsProductionProtein IntronsPseudohypertrophic Muscular DystrophyReagentResearchResearch AssociateResearch PersonnelResearch ResourcesResearch TrainingResearchersResource SharingResourcesSafetySkeletal MuscleSpeedTechnologyTestingTherapeuticTherapeutic Clinical TrialToxic effectToxicitiesTrainingTraining and OutreachTransgenic OrganismsTranslational ResearchTranslational ScienceTutoring and OutreachVariantVariationVisitVoluntary MuscleWorkX-linked dilated cardiomyopathyX-linked muscular dystrophyX-linked recessive muscular dystrophyZeugmatographyadeno-associated viral vector deliveryadeno-associated virus deliveryadeno-associated virus mediated deliveryadenovirus mediated deliverybenign X-linked recessive muscular dystrophybiologicboyscardiac musclecareerchildhood pseudohypertrophic muscular dystrophyclassic X-linked recessive muscular dystrophyclinical interventionclinical therapycohortcomputer based predictionconferenceconventiondata interpretationdelivered with AAVdelivery with AAVdesigndesigningdevelopmentaleffective therapyeffective treatmentgene repair therapygene therapygene-based therapygenetic therapygenomic therapygraduate studentheart muscleheavy metal Pbheavy metal leadimmunogenicityimprovedinteininter-institutionalmeetingmeetingsmicro-dystrophinmicrodystrophinmild X-linked recessive muscular dystrophymini pigmini-swineminiswinemodel of animalmouse modelmurine modelmuscle dystrophynew drug treatmentsnew drugsnew pharmacological therapeuticnew therapeuticsnew therapynext generationnext generation therapeuticsnovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel therapeuticsnovel therapyonline computeroutreachpig modelpiglet modelporcine modelpost-docpost-doctoralpost-doctoral traineepre-clinical studypreclinical studypredictive modelingprogressive muscular dystrophy of childhoodpseudohypertrophic adult muscular dystrophypseudohypertrophic muscular paralysisrecruitresearch associatessuccesssummitswine modelsymposiasymposiumtherapeutic agent developmenttherapeutic candidatetherapeutic developmenttransgene deliverytransgenictranslation researchtranslational investigationtranslational studytrial regimentrial treatmentvectorweb based
Description preview
Overall Summary/Abstract
The major theme of the Seattle Wellstone center is to improve therapeutic approaches to muscular
dystrophies by identifying and overcoming the emerging new barriers to successful clinical trials in muscular
dystrophies. The specific aims and objectives are to breach the major barriers to successful therapeutic
clinical…
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