A team of researchers at Généthon, the laboratory created and financed by the Association Française contre les Myopathies (AFM) thanks to the French Téléthon donations, has succeeded in repairing the muscles of mouse models of Duchenne muscular dystrophy thanks to a gene therapy called ‘exon skipping’. The exon skip occurs during the intermediate phase between the gene and the protein at the moment of splicing, and allows production of a truncated but functional protein to be restored. This advance illustrates the progress made in gene therapy techniques over the last ten years. By now, these techniques have become very sophisticated and, by intervening directly on the message of the gene, they open new therapeutic prospects for genetic diseases.
This work was carried out by a Généthon team led by Olivier Danos and Luis Garcia (CNRS UMR 8115) in collaboration with researchers at the Cochin Institute in Paris. It is published by the magazine Science in its online edition Science Express.

 Press release exon skipping 04/11/2004 ( doc  )