Optimization of AAV Vector Genome Design for the Treatment of Motor Neuron Diseases

Time: 11:15 am
day: Conference Day Two


  • Overview of the AAV gene therapy on SMA and ALS
  • Optimized vector genome Improves the efficacy and safety of AAV9-mediated gene therapy for SMA
  • Optimized artificial miRNAs by AAV9 vectors drastically improve survival and function in ALS mice