Animal model of Angelman Syndrome

US11793177B2 · kind B2 · utility

Assignees

• University of South Florida · US
• The Regents of the University of California · US
• THE TEXAS A&M UNIVERSITY SYSTEM · US

Inventors

• Edwin John Weeber · Apollo Beach, US
• David J. Segal · Davis, US
• Henriette O'Geen · Davis, US
• Benjamin Pyles · Davis, US
• Scott Victor Dindot · College Station, US

Key dates

• Filing date: Oct 3, 2017
• Grant date: Oct 24, 2023
• Expiry date: Dec 27, 2037

Classification

• Technology area (CPC A): Human Necessities
• CPC primary: A01K2267/0356
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

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.