UNIVERSITY OF SOUTH FLORIDA;THE REGENTS OF THE UNIVERSITY OF CALIFORNIA;THE TEXAS A&M UNIVERSITY SYSTEM
发明人:
WEEBER, Edwin,SEGAL, David,O'GEEN, Henriette,PYLES, Benjamin,DINDOT, Scott
申请号：
WO2017US54992
公开号：
WO2018067608(A2)
申请日:
2017.10.03
申请国别(地区):
世界知识产权组织国际局
年份:
2018
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.
