Full-Length Dystrophin and Representative Micro-dystrophins
Full-length dystrophin contains an N-terminal domain (N), 24 spectrin-like repeats (R1 to R24), four hinges (H1 to H4), a cysteine-rich domain (CR), and a C-terminal domain (CT). ΔDysM3 is the first synthetic micro-dystrophin. Δ3990, ΔR4–23/ΔC and μDys5R are three micro-dystrophins currently in use in clinical trials. H2 is marked in orange to indicate that it compromises micro-dystrophin function in the mouse DMD model (see Banks et al.53 for details). R16 and R17 are marked in red to indicate that they are the nNOS-binding domain (see Lai et al.46, 98 for details).