Trans -acting factors may cause dystrophin splicing misregulation in BMD skeletal muscles

Abstract
We analyzed dystrophin alternative splicing events in a large number of Becker muscular dystrophy (BMD) affected individuals presenting major hot‐spot deletions. Evidence is shown that altered splicing patterns in these patients do not directly result from the gene defect but probably derive from modifications in trans‐ rather than cis‐acting factors. Several potential CUG‐binding protein 2 (CUG‐BP2) binding sites were found to be located in the dystrophin gene region encompassing exons 43–60 and CUG‐BP2 transcript analysis indicated that not only expression levels are increased in dystrophic muscles but also that different CUG‐BP2 isoforms are expressed. The possibility that CUG‐BP2 might have a role in dystrophin splicing regulation is discussed.