Activating internal ribosome entry to treat Duchenne muscular dystrophy

Nat Med. 2014 Sep;20(9):987-8. doi: 10.1038/nm.3677.

Abstract

Mutations in the DMD gene, encoding dystrophin, cause the most common forms of muscular dystrophy. A new study shows that forcing translation of DMD from an internal ribosome entry site can alleviate Duchenne muscular dystrophy symptoms in a mouse model.

Publication types

  • Comment

MeSH terms

  • Animals
  • Dystrophin / genetics*
  • Exons*
  • Humans
  • Muscular Dystrophy, Duchenne / genetics*
  • Protein Biosynthesis*
  • Protein Isoforms / genetics*

Substances

  • DMD protein, human
  • Dystrophin
  • Protein Isoforms