Scientists Develop a Primate Model for Autism by Genome-Editing

June 16, 2019

A China-U.S. joint research team has engineered macaque monkeys to express a mutation linked to autism and other human neurodevelopmental disorders, through the genome-editing system CRISPR. These monkeys showed some behavioral traits and brain connectivity patterns similar to those in humans with these conditions.

The study, published in Nature, was conducted by scientists from the Shenzhen Institutes of Advanced Technology (SIAT) of the Chinese Academy of Sciences (CAS), Massachusetts Institute of Technology, Sun Yat-Sen University and South China Agricultural University.

Autism Spectrum Disorders (ASD) are complex developmental disorders with a strong genetic basis. Scientists have identified hundreds of genetic variants associated with ASD, many of which individually confer only a small degree of risk. In this study, the researchers focused on one gene with a strong association, known as Shank3.

So far mouse models of Autism Spectrum Disorder have not been highly successful due to neural and behavioural differences from humans. While the mouse research remains very important, we believe that primate genetic models will help us to develop better medicines and possibly even gene therapies for some severe forms of autism.

Primate models are much closer to humans, particularly around brain structure. The decision-making pre-frontal cortex in non-human primates, for example, is well-developed, making those primates potentially ideal for simulating certain brain diseases. Scientist involved in the study, said the new model type could help develop better neurodevelopmental disorder treatments in the future.