Rett Syndrome as a Case Example of Gene Dosage Sensitivity: miRARE-mediated Feedback Regulation as a Possible Solution

Time: 9:10 am
day: Day Two


  • Overview of AAV9 gene therapy
  • Is AAV9 good enough?
  • When building your AAV, how will you control dosage?
  • Evaluating dose sensitivity strategies and efficacy data using Rett syndrome as a case study
  • What is the best formulation to yield better biodistribution?