grant
Overcoming limitations for AAV gene therapy
Organization UNIVERSITY OF WASHINGTONLocation SEATTLE, UNITED STATESPosted 7 May 2014Deadline 31 Aug 2028
NIHUS FederalResearch GrantFY20253-D3-Dimensional3DAAV deliveredAAV deliveryAAV vectorAAV-based deliveryAAV-based vectorAAV-based viral deliveryAAV-mediated deliveryActin-Activated ATPaseAddressAdeno-associated-virus-based deliveryAdverse ExperienceAdverse eventAnimal ModelAnimal Models and Related StudiesAnimal Muscular DystrophyAnimalsBiological MarkersBiopsyBlood CellsCapsidCardiacClinicClinicalClinical DataClinical ResearchClinical StudyClinical TreatmentClinical TrialsCommon Rat StrainsComplexConduct Clinical TrialsDNA TherapyDNA cassetteDNA mutationDataDepressed moodDevelopmentDiseaseDisease ProgressionDisorderDoseDucheneDuchenneDuchenne muscular dystrophyDuchenne-Griesinger syndromeDystrophinEllis-van Creveld (EvC) syndromeEnhancersEnzyme GeneEnzymesEvaluationFSHDFacioscapulohumeral AtrophyFacioscapulohumeral Muscular DystrophyFacioscapulohumeral Type Progressive Muscular DystrophyFasioscapulohumeral Muscular DystrophyGene DeliveryGene InactivationGene SilencingGene TranscriptionGene Transfer ClinicalGene therapy trialGenerationsGenesGeneticGenetic ChangeGenetic InterventionGenetic TranscriptionGenetic defectGenetic mutationGoalsHOX geneHeartHereditaryHomeo Box GenesHomeobox Family GeneHomeobox GenesHomeodoamin GeneHomeotic GenesHumanImmuneImmune reactionImmune responseImmunesImmunityIndustryInflammationInheritance PatternsInheritedInjectionsIntervening Protein SequenceKnowledgeLandouzy Dejerine muscular dystrophyLandouzy-Dejerine DystrophyLengthMR ImagingMR TomographyMRIMRIsMagnetic Resonance ImagingMeasuresMedical Imaging, Magnetic Resonance / Nuclear Magnetic ResonanceMessenger RNAMiceMice MammalsMiniature SwineMinipigsModelingModern ManMolecularMonitorMurineMusMuscleMuscle DiseaseMuscle DisordersMuscle TissueMuscle functionMuscular DiseasesMuscular DystrophiesMutationMyocardiumMyodystrophicaMyodystrophyMyopathic ConditionsMyopathic Diseases and SyndromesMyopathic disease or syndromeMyopathyMyosin ATPaseMyosin Adenosine TriphosphataseMyosin AdenosinetriphosphataseMyosinsNMR ImagingNMR TomographyNuclear Magnetic Resonance ImagingPathologyPatientsPeripheral Blood CellPhenotypePlayPost-Transcriptional Gene SilencingProtein IntronsProteinsPseudohypertrophic Muscular DystrophyPublishingRNA ExpressionRNA InterferenceRNA Interference TherapyRNA SilencingRNA interference therapeuticsRNA interference-based therapyRNAiRNAi therapeuticsRNAi therapyRNAi vectorRNAi-based therapeuticsRNAi-based therapyRatRats MammalsRattusReagentResearchRibonucleotide ReductaseRoleSequence-Specific Posttranscriptional Gene SilencingSeriesSerious Adverse EventSerotypingSevere Adverse EventSkeletal MuscleSystemT-CellsT-LymphocyteTestingTherapeuticToxic effectToxicitiesTranscriptTranscriptionTransgenesVariantVariationVoluntary MuscleX-linked dilated cardiomyopathyX-linked muscular dystrophyX-linked recessive muscular dystrophyZeugmatographyadeno-associated viral vectoradeno-associated viral vector deliveryadeno-associated virus deliveryadeno-associated virus mediated deliveryadeno-associated virus vectoradenovirus mediated deliverybenign X-linked recessive muscular dystrophybio-markersbiologic markerbiomarkercardiac musclechildhood pseudohypertrophic muscular dystrophyclassic X-linked recessive muscular dystrophyclinical applicabilityclinical applicationclinical interventionclinical relevanceclinical therapyclinical translationclinically relevantclinically translatabledata modelingdelivered with AAVdelivery with AAVdepresseddesigndesigningdevelopmentalenhancer cassetteexpression cassettegene cassettegene repair therapygene therapygene transfer trialgene-based therapygenetic cassettegenetic therapygenome mutationgenomic therapyheart musclehemodynamicshiPSChost responsehuman iPShuman iPSChuman induced pluripotent cellhuman induced pluripotent stem cellshuman inducible pluripotent stem cellshuman inducible stem cellsimmune system responseimmunogenicityimmunoreactionimmunoresponseimprovedinduced human pluripotent stem cellsinnovateinnovationinnovativeintegration cassetteinteinknock-downknockdownmRNAmicro-dystrophinmicrodystrophinmild X-linked recessive muscular dystrophymini pigmini-swineminiaturizeminiaturizedminiswinemodel of animalmodel of datamodel the datamodeling of the datamouse modelmurine modelmuscle dystrophymuscle engineeringmuscularmuscular disordernew approachesnext generationnovelnovel approachesnovel strategiesnovel strategyoverexpressoverexpressionpatient screeningpig modelpiglet modelporcine modelprogressive muscular dystrophy of childhoodpromoterpromoter cassettepromotorpseudohypertrophic adult muscular dystrophypseudohypertrophic muscular paralysisreporter cassetteresistance cassettesadnessselectable cassetteselection cassetteserious adverse experienceserious adverse reactionsmall moleculesocial rolestop cassetteswine modelsynergismtherapeutic candidatethree dimensionalthymus derived lymphocytetooltranscription cassettetranscriptional cassettetranscriptional silencingtransgenetransgene cassettetransgene expressiontranslational studytrial regimentrial treatmentvector
Description preview
Gene therapy is a promising treatment for the muscular dystrophies. Our Center has played major roles in
advancing the use of AAV for muscle disease. These include extensive characterization of dystrophin and DUX4
expression and function, the discovery that AAV can be used for systemic gene delivery to muscle, and
generation of numerous small,…
🔒
Start 7-day free trial — $29.99/mo →Full details available on the Agency plan
Unlock the complete grant description, eligibility criteria, contract value, evaluation details and apply link — plus alerts, pipeline tracking, and CSV export.
Agency Plan
7-day free trialUnlock procurement & grants
Upgrade to access active tenders from World Bank, UNDP, ADB and more — with email alerts and pipeline tracking.
$29.99 / month
- 🔔Email alerts for new matching tenders
- 🗂️Track tenders in your pipeline
- 💰Filter by contract value
- 📥Export results to CSV
- 📌Save searches with one click