In-vivo base editing in a mouse model of autism, and more

Here is a roundup of autism-related news and research spotted around the web for the week of 23 February.

By Jill Adams

24 February 2026 | 2 min read

https://doi.org/10.53053/BRTA9097 Cite this article

Gene fix: In-vivo base editing can mitigate autism-like effects in mice carrying a variant of the CHD3 gene, according to a new paper. The variant, found in people with Snijders Blok-Campeau syndrome, causes altered sociability, cognition and motor coordination in mice. A TadA-embedded adenine base editor, delivered brain-wide via an adeno-associated virus, restored CHD3 protein levels and ameliorated the behavioral effects. The findings, the researchers write, “demonstrate that precise single-base correction in the postnatal brain can restore protein dosage and function, thereby offering a framework for the treatment of monogenic neurodevelopmental disorders.”

Research image of CHD3 protein levels in mice brains. — **Replacement parts:** Mice carrying a CHD3 variant have lower CHD3 protein levels (middle panels) than wildtype mice (left panels) in two brain regions (hippocampus, top row; somatosensory cortex, bottom). In-vivo base editing restores the protein (right panels).

Autism research spotted this week:

“Characterizing features of the genetic architecture underlying autism from a multi-ancestry perspective” medRxiv
“Up-regulation of Minibrain/DYRK1A contributes to macrocephaly and brain overgrowth in a Drosophila model of fragile X syndrome” Proceedings of the National Academy of Sciences
“Genome-wide significance reconsidered: Low-frequency variants and regulatory networks in autism” medRxiv
“Deficiency of the histone lysine demethylase KDM5B causes autism-like phenotypes via increased NMDAR signalling” bioRxiv