Gene therapy for muscular dystrophy: Lessons learned and path forward

► Molecular therapeutic approaches to treat muscular dystrophies are discussed. ► Exon skipping targets pre-mRNA allowing one or more exons to be omitted. ► Proof of principle for readthrough of stop codons has been established in DMD. ► Mini-dystrophin gene transfer resulted in an immune response, an unexpected outcome. ► Alpha-sarcoglycan gene transfer showed sustained gene expression for six months.