Team Led by Monkol Lek Advances Past Pre-IND Phase with DMD Gene Therapy
Duchenne Muscular Dystrophy (DMD) was first documented in the 1860s. More than a hundred years later, researchers discovered the genetic mutation underlying the progressive muscle degeneration that defines this disorder. Despite further studies into the mechanism of DMD, no effective treatment currently exists. Like many rare genetic diseases, DMD ultimately results in loss of quality of life and death. But thanks to advances in gene editing technology, that could soon change.