Focused ultrasound controls targeted gene editing in mice

In mice, researchers tested a gene-editing system that they activated from outside the body with focused ultrasound. They engineered compact gene-editing proteins that could be switched on with light. The system included an RNA guide that directs the protein to a DNA target. It could be delivered in one adeno-associated virus particle, a vehicle for carrying genetic material into cells.

The researchers combined focused ultrasound with mechanoluminescent particles, which emit light when mechanically stimulated, to activate the system. They reported successful gene editing in targeted skeletal muscle and brain regions. The method allowed them to control where and when editing took place in mice.