Evaluating the Impact of Genetic Diversity and Immunogenicity on the Efficacy of AAV Gene Therapy

Time: 1:30 pm
day: Day 1 Track A PM


  • Outlining key reasons for the lack of predictive murine models
  • Using genetically diverse collaborative cross (CC) mice to elucidate drug toxicity and host-pathogen factors that lead to infection persistence or clearance
  • Administering an AAV8 vector to 16 strains of CC mice and evaluating the effects of genetic diversity and adaptive immune responses on efficacy