The Texas A&M University System;University Of South Florida;The Regents of the University of California
发明人:
WEEBER, Edwin, John,SEGAL, David, J.,O'GEEN, Henriette,PYLES, Benjamin,DINDOT, Scott, V.
申请号:
EP17859048
公开号:
EP3518669A4
申请日:
2017.10.03
申请国别(地区):
EP
年份:
2020
代理人:
摘要:
The present invention concerns non-human animals with cells having a genome that is lacking the entire E3 ubiquitin ligase (Ube3a) gene (including all isoforms and alternative promoters). These animals are useful for modeling Angelman Syndrome. The invention also includes methods for assessing the effect of an agent, such as potential therapeutics, on an animal model by exposing the animal or cells, tissues, or organs isolated therefrom, to an agent of interest.