Efficacy of Gene Therapy Using a Dual Route of Administration in a Large Animal Model of Batten Disease

Time: 3:25 pm
day: Day One


  • A large animal model of disease is more clinically translational due to the brain structure and size and CSF flow
  • Selecting the right delivery route is critical for efficacy in alarge animal model
  • A dual route of administration was essential for targeting the major phenotypes characteristic of CLN5 Batten disease