Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

Nat Rev Genet. 2013 Jun;14(6):373-8. doi: 10.1038/nrg3460. Epub 2013 Apr 23.


Duchenne muscular dystrophy (DMD) is a devastating progressive disease for which there is currently no effective treatment except palliative therapy. There are several promising genetic approaches, including viral delivery of the missing dystrophin gene, read-through of translation stop codons, exon skipping to restore the reading frame and increased expression of the compensatory utrophin gene. The lessons learned from these approaches will be applicable to many other disorders.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Clinical Trials as Topic
  • Dependovirus / genetics
  • Dystrophin / chemistry
  • Dystrophin / genetics
  • Dystrophin / metabolism
  • Gene Expression
  • Genetic Therapy*
  • Genetic Vectors
  • Humans
  • Muscular Dystrophy, Duchenne / genetics
  • Muscular Dystrophy, Duchenne / therapy*
  • Mutation
  • Protein Structure, Tertiary
  • Utrophin / genetics
  • Utrophin / metabolism


  • Dystrophin
  • UTRN protein, human
  • Utrophin