Grant: China-Canada Joint Health Research Initiative

Duchenne muscular dystrophy (DMD) is one of the most common lethal genetic disorders worldwide. Currently, a most promising therapeutic option for DMD is exon skipping therapy. Exon skipping employs short DNA-like molecules like "Band-Aids" and target the mutated part of the gene. However, exon skipping faces three major challenges; 1) limited efficacy in the heart, 2) limited applicability (only 10% of DMD patients can be treated with exon skipping), 3) unknown stability and function of resulting proteins produced by the DNA Band-Aids. We will address all three challenges. Our research team in China (Dr. Haifang Yin) has developed novel DNA-like molecules that efficiently target both muscle and heart. Our research team in Canada (Dr. Toshifumi Yokota) has developed a cocktail of DNA Band-Aids that is applicable to majority of DMD patients and that can optimize the resulting proteins. In addition, the Canada team has developed a novel pig model of Duchenne muscular dystrophy for development of new drugs for DMD. The efficacy and safety of these novel molecules will be tested in our pig model. Our study will eventually lead to novel clinical trials for DMD.