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Autism Speaks Advances Animal Models

In partnership with SAGE Labs, Autism Speaks is developing new genetic rat models for studying autism spectrum disorders (ASD). The models will become important new tools for investigating the causes and treatment of autism.

The genetically engineered rat models of autism will be the first made widely available to autism researchers. Past genetic animal models of autism have involved mice. Rat models offer several advantages. “Rats display more social behaviors than mice, and are better at performing cognition tests," explains Daniel Smith, Ph.D., Autism Speaks senior director of discovery neuroscience. “Combine that with their larger brain size, and we now have models that make it easier to study the brain systems controlling behaviors that are altered in autism. These are important distinctions for modeling autism and how experimental treatments might work.”

Just as important, Dr. Smith adds, pharmaceutical companies regularly use rats for mandatory safety tests. So the new rat models could help streamline the medications discovery process. In addition, Autism Speaks investigators will be able to purchase these models at half their market price.

“Having another model species could accelerate our understanding of how genes associated with autism affect brain development and provide additional tools for identifying promising new drugs,” says Richard Paylor, Ph.D., professor of molecular and human genetics at Baylor College of Medicine. Dr. Paylor will be evaluating the new animal models, which will be created using technology developed by SAGE Labs.

The process deletes, or “knocks out,” specific genes in rat embryos. In humans, these genes are associated with autism. This produces animal models that exhibit autism-like behaviors caused by the gene change. These include abnormal social behavior, repetitive behaviors and decreased vocalizing. The science staff at Autism Speaks is currently working with scientists at SAGE Labs to determine which gene deletions will make the best models.

“By investing in new tools that will accelerate scientific discovery, we are preparing the grounds for a speedier development of drugs that could treat core autism symptoms,” says Rob Ring, Ph.D., Autism Speaks vice president of translational research. “Not only are we helping make these tools available to all autism researchers, but our investigators will doubly benefit by having cheaper access.”

For more perspective, please also see Dr. Paylor's blog.

Art credit: SAGE Labs, a division of Sigma Life Sciences