A study published inMolecular Therapy(2017) reported systemic delivery of CRISPR/Cas9 using adeno-associated virus (AAV) vectors in a canine model, restoring dystrophin expression in various muscles, including the heart, a key target given the cardiomyopathy associated with DMD.2Similarly, pigs were used to model DMD due to their physiological similarity to humans, with CRISPR/Cas9 successfully restoring dystrophin expression across multiple muscle types.19 The gene discussed is DMD; the disease is Duchenne muscular dystrophy.