Systemic Metachromatic Leukodystrophy Gene Therapy Leading to Disease Prevention in Arsa Ko Mice

Time: 2:00 pm
day: Day One: Track B PM


  • Biodistribution results will be presented from studies carried out in juvenile NHP and pig models
  • Comparison of lumbar IT vs ICM delivery routes as well as constitutive vs neuronal specific promoters in NHP
  • Optimization of factors such as dose volume, flush volume, and physical positioning