New therapies for muscular dystrophy: cautious optimism

Abstract

The quest for a therapy for muscular dystrophy has been the driving force behind the past 40 years of advances in this field. Numerous results, such as the identification of satellite cells and gene mutations that are responsible for most forms of dystrophies, advances in gene transfer and modification technology and, more recently, stem cells, have fueled hopes. However, administering corticosteroids still remains the only effective treatment available. Several recent advances have uncovered a diversity of possible therapeutic approaches, from pharmacological treatments to gene therapy (exon-skipping and adeno-associated viruses) and cell therapy with different types of newly identified stem cells. Importantly, a combination of these strategies might greatly enhance the possibility of successful therapy.