Duchenne muscular dystrophy (DMD) is an incurable, hereditary disease in which the muscles of those affected are gradually replaced by connective tissue. This leads to progressive muscle weakness. The cause of the disease is the absence of a large muscle protein called dystrophin. The Duchenne Foundation has been funding research on developing therapies and improving the lives of those affected for more than ten years.
This Research Award honours an idea that aims at converting connective tissue cells (fibroblasts) into functional muscle cells. For this purpose, molecular biologist Dr. Alf Spitschak as part of Prof. Dr. Brigitte M. Pützer's research group at the Institute of Experimental Gene Therapy and Cancer Research (IEGT) Rostock has developed adenovectors. These adenovectors can target specific cells and reprogramme them with high efficiency. Together with the doctoral candidate, Dinis Faustino, from the same research group, they succeeded in growing muscle fibres (myotubes) in cell cultures from fibroblasts. The obtained muscle cells are then characterised at the laboratory of physiologist Prof. Dr. Heinrich Brinkmeier. At University Medicine Greifswald’s Institute of Pathophysiology, he works together with medical doctoral candidates and Dr. Yaxin Zhang. Among other things, the research group will examine the myotubes’ electrophysiological properties and their calcium concentration. The plan is to treat a DMD-like disease in mouse models in the future. This will also include the restoration of functional dystrophin and the characterisation of the animals' muscle structure in detail. The study aims to ensure long-term muscle regeneration in muscular dystrophy.
Awarding the prize to the two researchers does not only promote an innovative project but also the interdisciplinary collaboration between the two research groups from Rostock and Greifswald.
Prof. Dr. Heinrich Brinkmeier
Institute of Pathophysiology
University Medicine Greifswald
Tel.: +49 3834 86 8100
Dr. Alf Spitschak
Institute of Experimental Gene Therapy and Cancer Research
Biomedical Research Centre Rostock
Schillingallee 69, 18057 Rostock
Tel.: +49 0381 494 4958