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?