Preclinical Studies in Large Animal Models Provide Insights Into AAV Integration & the Potential for Genotoxicity

Time: 5:15 pm
day: Day One


  • Hemophilia A dogs treated with AAV vectors delivering the factor VIII transgene
    were followed for as long as 10 years after vector administration providing a unique opportunity to study AAV integration
  • AAV integration studies in the hemophilia A dogs showed patterns of integration and clonal expansions containing integrated vector were detected in the absence of any evidence of tumorigenesis
  •  The studies highlight the importance of long term follow up in clinical studies