New targeted base-editing tool corrects genetic brain disorder in mice

Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized gene-editing tool, the TadA-embedded adenine base editor (TeABE), was delivered via a harmless modified virus to correct a CHD3 gene mutation in a mouse model.