It was also previously demonstrated in a mouse model of limb-girdle muscular dystrophy-2D that the use of a muscle-specific synthetic promoter, such as the Spc5.12 promoter21 used to restrict transgene expression in the present study, can lead to long-lasting transgene expression, whereas immune rejection was observed when the transgene is driven by the ubiquitous CMV promoter54. This evidence concerns the gene PCSK4 and limb-girdle muscular dystrophy.