Autism Speaks and SAGE Labs, a division of Sigma Life Science, this week announced a partnership to create genetically modified “knockout” rats to aid identification of new therapeutic approaches for the treatment of autism. The models, which will become available next year, are expected to simulate various symptoms and pathologies of autism spectrum disorders.
Animal models of a human disorder are an integral piece in the development of new treatment approaches. Existing 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 more limited use to researchers seeking to further understand the disorder or to identify new therapeutic approaches for its treatment. As rats are more physiologically similar to humans than mice are and have a much more diverse behavioral repertoire, 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.
“One of the handicaps to developing new therapeutics has been the lack of a really good system in which to model autism, determine what factors influence its development, and manipulate its biology,” stated Dr. Geri Dawson, Autism Speaks Chief Science Officer. “We have to make useful models available to researchers and pharmaceutical companies as rapidly as possible, which is why this new collaboration with SAGE labs is so important.”
Until recently it has been impossible to create rat models in which genes have been deactivated, but last year scientists at SAGE Labs developed the ability to produce models that may mimic the symptoms of a disorder by targeting specific genetic risk factors. Through the new collaboration with Autism Speaks, among the first animals to be developed with the technique will now 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 will 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, so the hope is that these models will aid directly in the design of new therapeutics.