NEW SUCCESSES IN MICE WITH MUSCULAR DYSTROPHY

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NEW SUCCESSES IN MICE WITH MUSCULAR DYSTROPHY
31 March 2007 - University of Bonn (Universität Bonn)

The diagnosis 'muscular dystrophy' is usually tantamount to a death sentence for those affected. One in three thousand male babies suffer from this incurable hereditary disease. The progress of the disease can only be slowed down through physiotherapy and medication. Scientists at Bonn University and at Pittsburgh Children's Hospital (USA) have now isolated a specific type of stem cell which can improve the regeneration of damaged muscle cells in mice suffering from muscular dystrophy.

The teams working with US researcher Dr. Johnny Huard and the Bonn physiologist Professor Anton Wernig were able to isolate a specific type of adult stem cell from the muscles of mice. The cells can replace degenerate muscular tissue in mice suffering from muscular dystrophy, doing this better than has hitherto been possible with other cells. The stem cells raise hopes of more effective therapies for degenerative muscular diseases like Duchenne's muscular dystrophy.

Up to now in transplants of muscle cells scientists have primarily had to contend with the low survival and reproduction rates of the available cell lines; what is more, the transplant generally triggered a persistent immune response, which eventually led to rejection of the new cells. 'However, we should not overrate these findings,' Professor Wernig warns. 'The cell type discovered in the mouse has not yet been proved to exist in humans.' However, he added that investigations along these lines were already being carried out as part of an EU project.

It is almost exclusively men who contract MD; as a consequence of a genetic defect those affected by this disease cannot synthesise important proteins needed by the muscle's metabolism. The muscle tissue progressively decomposes so that, in most cases, children are forced to use a wheelchair before they are ten years old. Finally the respiratory muscles and the myocardium are also affected. Patients die of cardiac insufficiency or dyspnoea.

Apart from the significance for muscle regeneration, the cell type detected appears to have wider ranging characteristics. 'In contrast to normal muscle stem cells from mice these stem cells are pluripotent,' Professor Wernig explains. 'They can also develop into different types of tissue, e.g. cartilage cells.' The cells do not lose their promising features even after the cells have divided several times. It has also been possible to isolate such pluripotent adult stem cells from other types of tissue, especially from bone marrow. Apart from embryonal stem cells they are regarded as the great white hope for future organ replacements.

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