Two years after the Round Table of January 2004 which concluded with the possibility of utilizing exon skipping as a therapeutic strategy, the clinical potential of this strategy has been confirmed and has been translated into different research approach

Two years after the Round Table of January 2004 which concluded with the possibility of utilizing exon skipping as a therapeutic strategy, the clinical potential of this strategy has been confirmed and has been translated into different research approaches (molecular, pharmaco-chemical, gene therapy, autologous cell therapy) which have all attained pre-clinical status today. Several clinical trials are already in preparation, but in parallel, basic research remains to be done in order to answer some unresolved questions (such as the immune response, systemic injection, production of batches of drugs). The international community which is concerned by Duchenne Muscular Dystrophy is mobilizing to cross a crucial frontier: at the initiative of the Parent Project USA an international consortium has been created and was presented for the first time in Monaco on January 15, 2006, with the goal of raising funds to finance clinical trials in Duchenne Muscular Dystrophy utilizing the promising technique of exon skipping.