Partnership to Create Genetically Engineered Knockout Rats for Improved Animal Models of Autism

Sigma Life Science announces a partnership between its SAGE Labs division and Autism Speaks to create genetically engineered knockout rats to aid identification of new therapeutic approaches for the treatment of autism. Combining the extensive knowledge of Autism Speaks with SAGE Labs’ proprietary SAGEspeed™ model creation process is expected to create a number of knockout rat models that simulate various symptoms and pathologies of autism.

Existing animal models of autism are mainly based on mice and, in many cases, do not adequately reflect the symptoms or biology of the disorder, making them of limited use to researchers seeking to further understand the disease or to identify new therapeutic approaches for its treatment. As rats are more physiologically and behaviorally similar to humans, and have a much more diverse behavioral repertoire for assessing learning and social behavior, they have become an important species for research in a number of fields and may serve as a more relevant organism in which to model autism and its related disorders.

Until recently, it has been impossible to create knockout rat models in which targeted genes have been deactivated. Scientists at SAGE Labs – using Sigma’s CompoZr® zinc finger nuclease (ZFN) technology as part of its SAGEspeed model creation process – now have the ability to produce knockout models designed specifically to mimic the symptoms of autism by targeting genes thought to be implicated in the disorder. Through the collaboration, among the first animals to be developed with the technique will be models for investigation of autism and its related disorders, including models of Fragile X and Rett syndromes, and mutations in neuroligin and neurexin genes. This is expected to enable researchers in the worldwide autism community to drive forward basic research into the biological mechanisms of disease. Most importantly, rats are currently also the preferred preclinical model for drug discovery research and development.

“We were particularly interested in partnering with SAGE because of the need to further our ability to carry out translational research in autism. More model systems are sorely needed,” said Dr. Sophia Colamarino, Vice President of Research for Autism Speaks. “Autism Speaks has direct connections to autism researchers across the U.S. and Europe. Combining this specialized knowledge with Sigma Life Science’s expertise and unique technology will result in significant benefits for researchers and hopefully a faster testing and adoption of these new models.”

“Sigma’s message, ‘Where Bio Begins’, is exemplified by the Company’s drive to further research in the field of autism by developing tools and animal models not previously available to the research community,” added Dr. Edward Weinstein, Director of SAGE Labs at Sigma Life Science. “Working in partnership with Autism Speaks, the leading organization in science, awareness and advocacy of autism-related issues, will provide the ideal opportunity to create a platform of very useful animal models for the research community. This exciting development should be hugely beneficial to researchers seeking to improve the lives of people with autism.”