Sigma Life Science's Sage Labs division and Autism Speaks are to create genetically engineered knockout rats to aid identification of new therapeutic approaches for the treatment of autism.
Combining the 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 behavioural repertoire for assessing learning and social behaviour, 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 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.
Rats are currently also the preferred preclinical model for drug discovery research and development.
