A China-U.S. joint research team has made new breakthroughs in developing non-human primate models for autism research, paving the way for possible drug discovery and gene therapy of the disorder.
The research was conducted by scientists from the Shenzhen Institutes of Advanced Technology under the Chinese Academy of Sciences, Massachusetts Institute of Technology (MIT), Sun Yat-Sen University and South China Agricultural University.
Autism is a neurodevelopmental disorder with a strong genetic basis. Scientists have identified hundreds of genetic variants associated with autism, and researchers in the study focused on one gene with a strong association called SHANK3.
Through the genome-editing system CRISPR, they engineered macaque monkeys with SHANK3 mutations and these monkeys showed some behavioral traits and brain connectivity patterns similar to those in humans with these conditions.
The primate model will provide a basis for a deeper understanding of the neurobiological mechanisms of autism and the development of more transformative therapeutics, according to the research team.
The research was published in the journal Nature.
