grant
Determinants of AAV Tropism
NIHUS FederalResearch GrantFY2026AAV vectorAAV-based vectorAdeno-Associated VirusesAdvisory CommitteesAffectBiodistributionBiologyBody TissuesCRISPRCRISPR/Cas systemCapsidCapsid ProteinsClinicalClinical TrialsClustered Regularly Interspaced Short Palindromic RepeatsCoat ProteinsCommunicationDNA RecombinationDNA TherapyDNA mutationDependoparvovirusDependovirusDoseE1A Binding Protein p300EP300EP300 geneEngineeringEpigeneticEpigenetic ChangeEpigenetic MechanismEpigenetic ProcessEvolutionExpression SignatureFamily suidaeFood and Drug AdministrationFundingFutureGene ExpressionGene Expression ProfileGene TranscriptionGene TransferGene Transfer ClinicalGenetic ChangeGenetic InterventionGenetic RecombinationGenetic TranscriptionGenetic defectGenetic mutationGenomeGoalsHepaticHost FactorHost Factor ProteinImmuneImmunesIntegration Host FactorsIntravenousKAT3BKidneyKidney Urinary SystemLibrariesLiverM mulattaM. mulattaMacacaMacaca mulattaMacaca rhesusMacaqueMapsMediatingMedicalMiceMice MammalsModelingMonkeysMurineMusMutationNatureOrphan DiseasePigsRNA ExpressionRare DiseasesRare DisorderRecombinationReporter GenesReportingRepressionRhesus MacaqueRhesus MonkeyRoleSafetySerious Adverse EventSerotypingSevere Adverse EventStructureSuidaeSwineSystemTask ForcesTherapeuticTissuesToxic effectToxicitiesTranscriptTranscriptionTranscriptional ControlTranscriptional RegulationTransgenic MiceTransgenic OrganismsTranslatingTreatment EfficacyTropismUSFDAUnited States Food and Drug AdministrationVariantVariationViral Coat ProteinsViral Outer Coat ProteinWorkadeno associated virus groupadeno-associated viral vectoradeno-associated virus vectoradvisory teambase editorclinical translationclinically translatabledesigndesigningdosageepigeneticallygene expression patterngene expression signaturegene repair therapygene therapeuticsgene therapygene-based therapeuticgene-based therapeuticsgene-based therapygenes therapeuticgenes therapeuticsgenetic therapygenome editinggenome mutationgenomic editinggenomic therapyhepatic body systemhepatic organ systemhistone acetyltransferase p300histone modificationimmunogenicityimprovedin vivointervention efficacymeetingmeetingsmouse modelmurine modelmutantnon-human primatenonhuman primatenovelorphan disorderp300porcinepre-clinicalpreclinicalrenalserious adverse experienceserious adverse reactionsocial rolestemsuidtherapeutic efficacytherapeutic genetherapy efficacythrombotictraittranscriptional profiletranscriptional signaturetransduction efficiencytransgene expressiontransgenicvectorvector controlvector genomevirus tropism
Description preview
Abstract
Adeno-associated virus (AAV)-based gene therapies have shown significant promise for the treatment of rare
diseases with unmet medical needs. In recent years, however, multiple reports of treatment-related serious
adverse events have been reported in several clinical trials. A recent U.S. Food and Drug Administration (FDA)
advisory…
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