Recently it was discovered that very short pieces of genetic material – so-called antisense oligonucleotides (AOs) – could alleviate the damaging effects of dystrophin mutations and restore the production of dystrophin protein to affected muscle. This they are able to do by a process called ‘exon skipping’, in which parts of the genetic code affected by the mutation are obscured in such a way that the code can be correctly read once again. The ability of AOs to restore dystrophin protein to muscle cells affected by dystrophin mutations was convincingly demonstrated by injecting the AOs directly into the hind limb muscle of the mdx mouse, which lacks dystrophin due to the presence of a mutation in much the same way as DMD patients. This striking effect has been recently confirmed in DMD patients directly by a Dutch research group that injected AOs directly into a leg muscle. AOs therefore have significant potential as medicines for DMD patients.