Neurodevelopmental disorders such as autism significantly trouble doctors and countless families worldwide. A collaborative study led by Chinese scientific teams demonstrates that delivering a novel gene-editing tool can achieve efficient, on-target single-base correction within the brain. The result was published in the international journal Nature on Wednesday.

The researchers looked at Snijders Blok–Campeau syndrome (SNIBCPS), which is caused by pathogenic variants in CHD3 and manifests with intellectual disability, autistic-like behaviors and difficulties in moving the body. They engineered a TadA-embedded adenine base editor (TeABE) and delivered it into the brains of mice with abnormal CHD3 levels. After the treatment, the mice regained normal behaviors and cognitive abilities, with CHD3 levels returning to the normal range. Other nearby genes were not affected. The findings establish in vivo base editing as a viable therapeutic approach for CHD3-related neurodevelopmental disorders.

The scientists also tested the translational feasibility in nonhuman primates, which showed results consistent with those in mice.

The researchers acknowledged that there is still a long way to go from animal models to clinical trials. The research team is already planning the next phase of experiments.