Molecular targets to treat muscular dystrophies

Swiss Med Wkly. 2014 Feb 19;144:w13916. doi: 10.4414/smw.2014.13916.


Muscular dystrophies are classically subdivided according to their clinical phenotype, and were historically defined as progressive myopathies in which muscle biopsies demonstrate muscle fibre necrosis and regeneration, as well as replacement of muscle fibres by adipose and connective tissue. In recent years, great progress has been made in identifying the genetic basis of many myopathies, thereby presenting opportunities to develop therapeutic strategies that act on specific molecular pathomechanisms. The different therapeutic strategies and their molecular targets will be reviewed.

Publication types

  • Review

MeSH terms

  • Animals
  • Dystrophin / genetics*
  • Genetic Therapy*
  • Humans
  • Molecular Targeted Therapy*
  • Muscular Dystrophies / genetics
  • Muscular Dystrophies / therapy*
  • Muscular Dystrophies, Limb-Girdle / genetics
  • Muscular Dystrophy, Facioscapulohumeral / genetics
  • Myoblasts / transplantation
  • Myotonic Dystrophy / genetics


  • Dystrophin