In-vivo base modifying in a mouse mannequin of autism, and extra
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.”
