Fig. 3. The principles of surrogate gene therapy, with μUtro as an example.

Surrogate microgene therapy can improve muscle function and structure. In comparison to dystrophin, μUtro also encodes the actin-binding domain (ABD1). This microgene is effective despite lacking the C-terminal (CT) domain and having only three hinge (H) domains, four spectrin-like repeats (R) and the cysteine-rich (CR) domain. (Adapted from Davies and Chamberlain [41] with permission).