[China Tech] Local Experts Develop Novel Gene-editing Tool for Autism

China Tech is a column dedicated to the innovations reshaping China – and, inevitably, the world. From cutting-edge AI labs and next-gen robotics to homegrown apps that redefine daily life, we explore the breakthroughs that emerge from the country's relentless drive for technological dominance. Some are game-changers, others cautionary tales, but all offer a glimpse into the future as it's being built, at breakneck speed, in China.

Researchers from Xinhua Hospital and Songjiang Hospital, both affiliated to the Shanghai Jiao Tong University School of Medicine, were looking to address the root cause of autism by accurately repairing the mutated genetic locus.

To achieve this, they engineered a novel TadA-embedded adenine base editor. Unlike conventional approaches that merely block the mutated sequence, this tool precisely identifies the erroneous base pair and restores it to the correct one without disrupting the overall DNA structure. This strategy minimizes the risk of unintended genomic alterations.

In animal trials, the gene-editing tool was administered to mice carrying CHD3 mutations. It successfully reached multiple brain regions and managed precise repairs without affecting adjacent normal genes. As CHD3 protein levels were restored, the mice showed significant behavioral improvements – including normalized social interaction, enhanced learning and memory, and recovered motor function.

The research team has since extended the trials to monkeys, with promising results observed thus far.

According to Dr Li Fei from Xinhua Hospital, a leading expert on the research team, her group has focused on autism studies for decades. They spent over ten years establishing the world's largest single-site cohort study on young autistic children, through which they identified CHD3 and related genes as important pathogenic factors in Chinese children with autism.

"So we decided to start from the CHD3 gene mutation to look for solutions for autistic children," she said. "The current success in animal tests has offered precious evidence for follow-up clinical research."

Officials from the School of Medicine noted that this breakthrough aligns with the institution's strategic focus on artificial intelligence-assisted medicine, brain science, immunology, and virology.

A research report on another of Xinhua Hospital's projects – an AI-assisted diagnostic tool for rare diseases – was also recently published in the renowned journal Nature. Through the integration of medicine and engineering, the school has established several research institutes dedicated to AI, immunotherapy, and virology to accelerate medical innovation and the development of biomedical engineering.

"We have adopted various measures to encourage and boost medical innovation to help solve clinical problems and eventually benefit patients," said Dr Fan Xianquan, director of the Shanghai Jiao Tong University School of Medicine.

"A pharmaceutical innovation hub has recently been established by our School of Medicine to speed up the transfer of clinical research into products and technologies that help patients sooner and more quickly."