The Translational Myology Laboratory (PIs Professor Thomas Voit and Dr. Julie Dumonceaux) is focused on the development of innovative treatment approaches for neuromuscular diseases. We use an Adeno-associated-virus (AAV) platform and muscle cell immortalization or iPS techniques to both create disease-relevant cellular and animal models as well as treatment approaches. In these we combine technologies of vector-mediated gene knock-down or gene transfer with other approaches aiming at stabilizing or correcting genetic abnormalities.
Our current work addresses treatment approaches for Facio-scalulo-humeral muscular dystrophy (FSHD), Myotonic Dystrophy, and Duchenne- Becker- and Limb girdle muscular dystrophy. In addition, we investigate general aspects of muscle homeostasis such as myostatin mechanisms and how these can be exploited for ameliorating wasting muscle conditions.