Cutting-edge Chinese gene-editing technique raises prospect of new autism treatments
Lab tests on mice given the treatment showed considerable cognitive and behavioural functions, new study says
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.