Gene therapy : Encouraging results in a Phase I clinical trial in limb-girdle muscular dystrophy type 2C

Evry (France), January 12, 2012 – The results of a Phase I clinical trial of gene therapy for limb-girdle muscular dystrophy type 2C (a rare neuromuscular disease) have just been published in the journal Brain on January 11, 2012. The trial started in December 2006 and has been sponsored by Généthon (the not-for-profit research lab created by the French Muscular Dystrophy Association (AFM) and which is funded almost exclusively by donations from France’s annual Telethon). The trial at Pitié-Salpêtriere (AP-HP) is being led by principal investigators Professor Serge Herson (Head of the Department of Internal Medicine1) and Professor Olivier Benveniste (Institute of Myology). The study’s primary objective was to evaluate the safety of local injection of increasing doses of an adeno-associated virus (AAV) vector harboring a “healthy” copy of the gene for gamma-sarcoglycan (the defective protein in this disease). Secondary objectives included the assessment of local and systemic immune reactions and the quality of gene transfer in the injected muscles in terms of efficacy, expression and distribution.