Non-invasive control of gene editing in vivo by photoswitchable Cas12f and focused ultrasound.
Non-invasive, precise gene editing in vivo achieved using a compact, photoswitchable Cas12f activated by focused ultrasound in mice.
- Why it matters: Controlling gene editing spatially and temporally within living tissues is crucial for therapeutic safety and efficacy, yet current methods lack non-invasive deep tissue activation and single AAV delivery.
- What they did: Researchers engineered a compact, photoswitchable Cas12f (psCas12f) compatible with single AAV particles and used focused ultrasound combined with mechanoluminescent particles to activate gene editing in specific tissues of mice.
- The result: This approach enabled successful, localized gene editing in skeletal muscle and brain regions without invasive procedures, paving the way for non-invasive, targeted gene therapies.