Periodic Reporting for period 1 - DMD2CURE (Correction of duplications in the DMD gene by a CRISPR/Cas9 approach)

Duchenne muscular dystrophy (DMD) is an X-linked recessive muscle-wasting disease, characterized by progressive weakening of skeletal, respiratory, and cardiac muscle followed by necrosis and fibrosis. DMD affects ~1:5000 live male births and is associated with delayed motor...