Dr. Ype Elgersma Erasmus University Medical Center Rotterdam, Netherlands
ASO Treatment for a Better Understanding of AS Pathophysiology and Optimizing Therapeutic Efficacy
Researchers will investigate in more detail the requirements of ASO-mediated rescue of behavioral phenotypes of an AS mouse model.
The most important questions they will attempt to address are:
- How much ASO and how much UBE3A protein is really needed to get a rescue of behavioral phenotypes?
- Is ASO treatment required throughout life?
- When does ASO treatment have to start to get full rescue of epilepsy in AS mice?