Evaluation of the rASO in the Rat Model of Angelman syndrome on the EEG and Seizure Phenotype | $163,700

The objective of this grant is to evaluate whether delivery of ASO designed to reactivate paternal ube3a in the AS rat improves the AS-related EEG and seizure phenotype. Preliminary analyses of the AS rat model indicate that EEG and seizure phenotypes are largely similar to those observed in the AS mouse model and AS patients.