Subsequently, the CRISPR array is transcribed into a long precursor crRNA, which is further processed into the mature crRNA by Csy4 protein. Finally, this stable Csy4-crRNA complex subsequently ...

In about a third of patients, the gene for dystrophin has small deletions or single base mutations that render it nonfunctional, which makes this gene an excellent candidate for gene editing.

Genome editing corrects the cardiac abnormalities in patient's engineered heart tissue. This material relates to a paper that appeared in the date, issue of Science Advances, published by AAAS.

In addition, first-generation CRISPR systems (Cas9 and Cas12a ... and heart muscle in a humanized mouse model of Duchenne Muscular Dystrophy (DMD), when delivered via a single AAV vector.