Chinese & U.S. Researchers Develop a Primate Model for Autism by Genome-Editing

Jun 13, 2019 | Autism, Primate Model

A joint China-U.S. research team reported the generation of germline-transmittable cynomolgus macaques with Shank3 mutations, known to cause a form of autism.

The Research Collaborative

The research sites included 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 Industry.

The Study

Through the genome-editing system CRISPR, they engineered macaque monkeys to express a gene mutation linked to autism and other neurodevelopmental disorders in human. These monkeys showed some behavioral traits and brain connectivity patterns similar to those in humans with these conditions.

Autism Spectrum Disorders (ASD)

Autism Spectrum Disorder is a 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 the study, the researchers focused on one gene with a strong association, known as Shank3.

Models

FENG Guoping of MIT noted “The new type of model, however, could help scientists to develop better treatment options for some neurodevelopmental disorders.”  Mouse models of ASD, due to their neural and behavioral differences from primates, haven’t worked out very well. The reported behavioral and neural traits of Shank3 mutant primates provide new insights into the circuit-based pathophysiological model of ASD.

The primate model is close to humans in evolution and had many similarities to humans in brain structure. For example, the prefrontal cortex in nonhuman primates is well developed, which plays important roles in decision-making, attention and social interactions.  Deficits in these cognitive functions have been associated with brain disorders including autism. Therefore, “nonhuman primates are hopeful to become an ideal animal model for stimulating some human brain diseases,” said professor ZHOU Huihui from SIAT.

“We urgently need new treatment options for autism spectrum disorder, and treatments developed in mice have so far been disappointing. 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,” says Robert Desimone,  the director of MIT’s McGovern Institute for Brain Research, the Doris and Don Berkey Professor of Neuroscience, and an author of the paper.

Association for Assessment and Accreditation of Laboratory Care (AAALAC)

A private nonprofit organization that promotes the humane treatment of animals in science through voluntary accreditation and assessment programs. SIAT received AAALAC accreditation in 2018 for its primate experiment platform, which laid a foundation for collaboration with international pharmaceutical companies to pursue new treatments of brain disorders in the future.

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