Use and production of CHD8+/− transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder

US11459557B2 · kind B2 · utility

Assignees

• The Broad Institute, Inc. · US
• Massachusetts Institute of Technology · US

Inventors

• Randall Jeffrey Platt · Cambridge, US
• Feng Zhang · Cambridge, US
• Ian Slaymaker · Cambridge, US

Key dates

• Filing date: Mar 23, 2017
• Grant date: Oct 4, 2022
• Expiry date: Mar 23, 2037

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2830/008
• WIPO field: Biotechnology
• WIPO sector: Chemistry

Abstract

Methods and compositions for inducing a plurality of mutations in transgenic Cas9 eukaryotes to model a neuronal disease or disorder. The invention further comprehends testing putative treatments with such models, e.g., testing putative chemical compounds that may be pharmaceutically relevant for treatment or gene therapy that may be relevant for treatment, or combinations thereof. The invention allows for the study of genetic diseases and putative treatments to better understand and alleviate a genetic disease or a condition, e.g., autism, autism-spectrum disease or disorder, obsessive-compulsive disorder, or psychiatric disorders.