Frontline Studies on Duchenne Muscular Dystrophy Treatment

Abstract

  Duchenne muscular dystrophy is the most common inherited muscular disease, characterized by progressive muscle wasting, commonly leading to death in the twenties due to cardiac or respiratory failure. In order to establish treatment for Duchenne muscular dystrophy, many kinds of studies including gene transfer strategies have been conducted, but no effective treatment has been established. Two plausible ways have been proposed. One is the induction of exon skipping to correct out-of-frame to in-frame dystrophin mRNA, thereby leading to the production of truncated dystrophin. The other one is the induction of read-through of nonsense mutation which ignores a premature stop codon to translate dystrophin mRNA into dystrophin. Here, these two ways are presented and discussed.