To date, direct delivery of the protein (35) or stabilization of the protein or RNA (36,37), viral approaches (38,39), non-viral strategies such as recombinant biglycan (40) and oral compound administration designed to modulate the utrophin expression at the transcriptional level (41,42), showed that maintaining utrophin expression in mdx muscle fibres could decrease the progression of the disease and represents a promising therapeutic avenue for DMD. The gene discussed is UTRN; the disease is Duchenne muscular dystrophy.