DMD and Duchenne muscular dystrophy: A sample case is a 27-year-old Duchenne Muscular Dystrophy (DMD) patient who was treated with recombinant adeno-associated virus (rAAV) serotype 9 dSaCas9 (‘dead’ Staphylococcus aureus Cas 9, with inactivated nuclease activity) fused to VP64, which, as a custom CRISPR-transactivator treatment, was designed to upregulate cortical dystrophin (Ref 196).