Gene therapy for muscular dystrophy: moving the field forward

Pediatr Neurol. 2014 Nov;51(5):607-18. doi: 10.1016/j.pediatrneurol.2014.08.002. Epub 2014 Aug 7.


Gene therapy for the muscular dystrophies has evolved as a promising treatment for this progressive group of disorders. Although corticosteroids and/or supportive treatments remain the standard of care for Duchenne muscular dystrophy, loss of ambulation, respiratory failure, and compromised cardiac function is the inevitable outcome. Recent developments in genetically mediated therapies have allowed for personalized treatments that strategically target individual muscular dystrophy subtypes based on disease pathomechanism and phenotype. In this review, we highlight the therapeutic progress with emphasis on evolving preclinical data and our own experience in completed clinical trials and others currently underway. We also discuss the lessons we have learned along the way and the strategies developed to overcome limitations and obstacles in this field.

Keywords: exon skipping; gene therapy; muscular dystrophy.

Publication types

  • Review

MeSH terms

  • Animals
  • Genetic Therapy / methods*
  • Genetic Therapy / trends*
  • Humans
  • Muscular Dystrophies / genetics*
  • Muscular Dystrophies / therapy*