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Dannie Peng

Chinese scientists have used a cutting-edge gene-editing tool to correct a DNA mutation responsible for cognitive and behavioural problems – a move that could eventually lead to the development of new autism therapies.
In lab tests, mice that had been engineered to have the mutation showed a striking change in their behaviour, such as the way they interacted with other mice, after being given an injection to introduce the edited genes.
The Shanghai-based research team is looking for ways to treat Snijders Blok‑Campeau syndrome, a rare neurodevelopmental condition.
The syndrome was first described in 2018 by Lot Snijders Blok, a scientist at Radboud University in the Netherlands, and Philippe Campeau, from the Baylor College of Medicine in Texas.
It is characterised by a range of symptoms, including developmental and speech problems, intellectual disability, distinctive facial features and structural brain abnormalities. It is also frequently accompanied by autism spectrum disorder.
Because of its complex origins and the difficulty of targeting the underlying mechanisms, there are no effective treatments, placing a heavy burden on affected children and their families.
The syndrome is caused by variants in a gene called CHD3, which plays a crucial role in early development and helps maintain DNA structure and integrity.
The researchers first introduced the genetic mutation into mice and then used a new CRISPR-based genome-editing tool to repair it.
They focused on two fundamental building blocks of DNA – known as the AT and GC base pairs – which hold together the DNA’s double helix.

According to the team, mismatches in the AT base pair cause a protein dysfunction leading to abnormal brain development.
They wrote that their new “craftsman” editor could precisely detect and correct the faulty AT base pair back to the normal GC base with a much lower risk of disrupting the overall DNA structure compared with conventional gene-editing techniques.
After an initial screening phase, the team delivered this bespoke gene editor into the mice via intravenous injection, triggering a series of striking changes.
Animals that had previously shunned their cage mates began actively interacting with them. In object recognition tests, they could clearly distinguish new objects from familiar ones, signalling an improvement in cognitive functions.
In the Barnes maze learning task, a long-established way of testing rodents’ spatial learning and memory, their performance returned to normal after being given the injections.
At the same time, their motor skills improved: muscle tone increased, and an unsteady, clumsy gait gave way to more coordinated movement.
“Behavioural experiments demonstrated notable improvements in key symptoms, including cognitive deficits, spatial learning and memory impairments, autistic-like behaviours and hypotonia [low muscle tone], which all underscore the efficacy of our approach.
“This intervention restored CHD3 levels and ameliorated behavioural abnormalities in vivo,” the authors wrote in their study, published in the peer‑reviewed journal Nature earlier this month.


The researchers moved beyond rodents and repeated some of the key aspects of the research in macaques to further test its potential.
They observed that the edited genes had a noticeable impact on the primates’ brains, increasing their confidence this approach could be translated to humans.
But they warned there was still a substantial journey from animal studies to therapies for humans, and said “bridging the gap between preclinical research and clinical translation remains a significant challenge”.
The work was led by Qiu Zilong from Shanghai Jiao Tong University school of medicine, Cheng Tianlin from the State Key Laboratory of Brain Function and Disorders at Fudan University, and Li Fei from Xinhua Hospital.


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Cutting-edge Chinese gene-editing technique raises prospect of new autism treatments

The scientists used a new gene-editing tool to correct the mutation in mice. Photo: Shutterstock

The mice showed marked differences in behaviour after the edited DNA was injected into them. Photo: Shutterstock 

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Lab tests on mice given the treatment showed considerable cognitive and behavioural functions, new study says


Lab tests on mice given the treatment showed considerable cognitive and behavioural functions, new study says.


Biomedicine: Science

Cutting-edge Chinese gene-editing technique raises prospect of new autism treatments

.css-1c6uqr6{color:inherit;font-weight:inherit;font-size:inherit;font-family:inherit;line-height:inherit;overflow-wrap:break-word;}Lab tests on mice given the treatment showed considerable cognitive and behavioural functions, new study says

Lab tests on mice given the treatment showed considerable cognitive and behavioural functions, new study says