Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

Personalized organoids — ‘mini-organs’ — may accelerate treatment for patients with rare genetic disorders. Courtesy of Europeana via Unsplash Across the globe, countless babies ...

NanoCas, an ultracompact nuclease, enables efficient muscle editing in NHPs using a single AAV - advancing gene editing for hard-to-target tissues.