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Stem Cells and Gene Therapy Used to Treat Muscular Dystrophy

A team of Italian researchers are hoping to combine the power of stem cells and gene therapy to develop a treatment to combat muscular dystrophy (MD). Dr Giulio Cossu of the Stem Cell Research Institute in Milan and the University of Rome, and colleagues have already managed to use stem cells to strengthen the muscles of mice with a form of MD, a genetic disease characterized by progressive weakness and degeneration of the muscles. There are a number of different types of MD – all of which are currently untreatable and incurable. Cossu worked with a type of stem cell known as a mesoangioblast. The researchers injected mesoangioblasts into the arteries of mice genetically engineered to lack the alpha sarcoglycan gene, which causes the mice to develop a form of MD. They then genetically engineered the mesoangioblasts by inserting a healthy copy of the alpha sarcoglycan gene into the cells. The engineered cells were then injected into the mice. Tests conducted three months later showed that healthy alpha sarcoglycan proteins were present in the muscles of the treated mice and that the animals had regained virtually all of their muscular strength.

SOURCE/REFERENCE: Reported by www.reutershealth.com on the 11th July 2003.

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