The idea for the development of splicing therapy for DMD to skip the exons with mutation on pre-mRNA transcripts derived from several observations on: (i) the modulation using antisense oligonucleotides of β-globin gene associated with β-thalassaemia; (ii) the presence of revertant fibers (dystrophin-positive) in DMD patients, due to intrinsic alternative splicing; and (iii) the shorter but functional dystrophin proteins found in the milder form of Becker muscular dystrophy (BMD) [65]. This evidence concerns the gene DMD and Becker muscular dystrophy.