Harnessing a Large Animal Model to Overcome Translational Barriers in Angelman Syndrome
Time: 9:00 am
day: Day 2
Details:
- Unpack why mouse models fail to recapitulate Angelman syndrome phenotypes and challenges associated with rodent models in preclinical development
- Rationale and characterization of the Angelman pig model, including phenotypic fidelity, regulatory implications, and translational value
- Target discovery in a complex genomic landscape: Exploring how integrated approaches in genetics and biology helped identify functionally relevant regions for therapeutic development for Angelman syndrome
