Funding Research for Treatments and a Cure

Neural circuits and behavior in mouse models of Ube3a disorders
← Back to Search

Michael Sidorov Michael Sidorov, PhD Children's National Research Institute

Neural circuits and behavior in mouse models of Ube3a disorders


As we move towards promising clinical trials for AS, a major challenge is deciding how to measure improvement after treatment. This study will apply mathematical approaches to data from new and existing behavioral tests using the Angelman syndrome mouse model to determine a single behavior score for each AS mouse that captures performance on all of the different behavior tests. The researchers will determine whether this score correlates with the established EEG biomarker in the AS mouse model and whether the score and biomarker improves when UBE3A is restored in the mouse model. 

Ultimately, this study will allow researchers to carefully compare different therapeutic approaches in the AS mouse model to determine which one works best. Also, it will provide an important proof-of-concept where this same approach may be applied to AS patient data before and after therapeutic treatment to generate better outcome measures for AS trials. 



An approach called “multidimensional analysis,” was developed.  This approach combines the results of many different behavioral tests and summarizes behavior as a single severity score. Using this severity score, will help labs and companies test the effectiveness of many different types of treatments preclinically to evaluate the best candidates to move forward towards clinical trials.

See the article in Translational Psychiatry