At the 16th annual meeting of the American Society of Gene & Cell Therapy, the Muscular Dystrophy Association (MDA) and the Association Française Contre Les Myopathies (AFM) hosted a joint symposium on advancing gene therapy for neuromuscular diseases. As noted by one of the symposium speakers, Jeffrey Chamberlain, finding effective methods of gene delivery to muscle tissue is one of the main challenges facing the field. He explained the current approaches to gene therapy and their downfalls. Other speakers discussed ideas specific to their own research, as well as more general concepts, including overcoming the delivery challenge. Molecular geneticist Xiao Xiao, reviewed his lab’s recent efforts at overcoming an important hurdle in gene therapy efforts: delivering sufficient numbers of therapeutic genes to all of the muscles that need them. At Généthon, research scientist Anna Buj-Bello presented updates about a collaboration with Martin Childers on an MDA-AFM-funded project to investigate gene therapy for myotubular myopathy (MTM), in which the team is using a canine research model that has a naturally occurring form of the disease. Fulvio Mavilio, an expert on gene therapy for rare diseases and director of Généthon, presented information about two gene therapy efforts under development for DMD, focussing in particular on an exon-skipping approach that has the potential to persist much longer in the body than exon-skipping strategies currently in testing. A clinical trial to test the new method is expected to begin in late 2013 or early 2014.
