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

People with certain mutations in the gene coding for dysferlin develop muscular dystrophy—a group of muscle wasting diseases ... Escobar uses CRISPR-Cas9, which is often described as "gene ...

Then Crispr came along—the elegant enzymatic apparatus ... vectors already provides the ability to achieve in vivo editing in eye, liver, and muscle, though there is still much work to be ...