Funding Research for Treatments and a Cure

ASO Treatment for a Better Understanding of AS Pathophysiology and Optimizing Therapeutic Efficacy
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Dr. Ype Elgersma Dr. Ype Elgersma Erasmus University Medical Center Rotterdam, Netherlands
2022

ASO Treatment for a Better Understanding of AS Pathophysiology and Optimizing Therapeutic Efficacy

$199,100

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:

  1. How much ASO and how much UBE3A protein is really needed to get a rescue of behavioral phenotypes?
  2. Is ASO treatment required throughout life?
  3. When does ASO treatment have to start to get full rescue of epilepsy in AS mice?