Neurodevelopmental disorders such as autism pose significant challenges for healthcare professionals and families worldwide. In a groundbreaking study led by Chinese researchers, a novel gene-editing technology demonstrates the ability to make precise single-base corrections within the brain. The findings of this study were published in the international journal Nature on Wednesday, highlighting advancements in therapeutic interventions for autism.

The research team focused on Snijders Blok–Campeau syndrome, or SNIBCPS, which results from pathogenic variants in the CHD3 gene. This condition is characterized by intellectual disability, autistic-like behaviors, and difficulties with bodily movement. To address this, the researchers developed a TadA-embedded adenine base editor, known as TeABE, and applied it to the brains of mice exhibiting abnormal levels of CHD3.

After treatment, the mice displayed restored normal behaviors and cognitive functions, alongside a significant return of CHD3 levels to within the normal range. Importantly, the treatment showed no adverse effects on neighboring genes, indicating the precision of the in vivo base editing approach. This evidence suggests that this method could be a viable therapeutic strategy for neurodevelopmental disorders linked to the CHD3 gene.

Furthermore, the scientists tested the translational feasibility of this technology in nonhuman primates. The results obtained were consistent with those achieved in mice, reinforcing the potential of this gene-editing method across species.

Despite these promising results, the researchers recognized that a substantial journey lies ahead from initial animal models to clinical trials. As a result, the research team is already strategizing the next phase of experiments to further investigate the application of this gene-editing technology.