grant
Adeno-Associated Virus Gene Therapy Vectors: Molecular Interactions on Cell Entry
Organization UNIVERSITY OF MISSOURI-COLUMBIALocation COLUMBIA, UNITED STATESPosted 1 Aug 2017Deadline 31 Jul 2028
NIHUS FederalResearch GrantFY20253-D3-Dimensional3DAAV vectorAAV-based vectorAdeno-Associated VirusesAlgorithmsAntibodiesAran-Duchenne diseaseBindingBiologic ModelsBiologicalBiological ModelsBlood SerumCell BodyCell NucleusCellsClinical TrialsComplexComputing MethodologiesCruveilhier diseaseCryo-electron MicroscopyCryo-electron tomographyCryoelectron MicroscopyDNA TherapyDNA mutationDependoparvovirusDependovirusDevelopmentDiseaseDisorderDoseEC 3.1.1.4Electron CryomicroscopyElectron MicroscopyEndosomesEngineeringExternal DomainExtracellular DomainFoundationsFreedomGene Therapy VectorsGene Transduction AgentGene Transduction VectorsGene Transfer ClinicalGenetic ChangeGenetic DiseasesGenetic InterventionGenetic defectGenetic mutationGlycansGoalsH-bondHumanHydrogen BondingImmune EvasionLecithinase A2LibertyLocationMapsMediatingModel SystemModern ManMolecularMolecular InteractionMutationNucleusPLA2Phospholipase A2PolysaccharidesPredispositionProteinsReceptor CellReceptor ProteinReceptosomesResolutionRetinal DystrophyRiskRoleSafetySerumSpecificitySpinal Muscular AtrophyStructureSumSusceptibilityTestingTherapeuticViraladeno associated virus groupadeno-associated viral vectoradeno-associated virus vectorbiologiccell transductioncellular transductioncomputational methodologycomputational methodscomputer based methodcomputer methodscomputing methodconformational conversionconformational transitioncryo-EMcryo-EM tomographycryoEMcryoEM tomographycryoelectron tomographycryogenic electron microscopydelivery vectordelivery vehicledesigndesigningdevelopmentalelectron cryo-tomographygene repair therapygene therapygene-based therapygenetic conditiongenetic disordergenetic therapygenome mutationgenome wide screengenomic therapyimmune evasiveimmunotoxicityimprovedin vivolecithinase Ananodiskneutralizing antibodyneutralizing mAbneutralizing monoclonal antibodiesopen sourcephosphatidasephosphatidolipasephosphatidylcholine 2 acylhydrolasepolyclonal antibodyprotonationprototypepublic health relevancereceptorreceptor bindingreceptor boundresolutionsrestraintsocial rolethree dimensionaltoxic reaction in immunologytransduced cellsvectorvirus host interaction
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Project Summary / Abstract
Adeno-associated virus (AAV) is a leading delivery vector for gene therapies of a wide range of genetic
disease and predilections. The FDA has recently approved AAV-mediated treatments for spinal muscular
atrophy (SMA) and RPE-associated retinal dystrophy, while 150 clinical trials are ongoing. However, inefficient…
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