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15 - 09 - 2016

Some News for gene therapy to treat Duchenne muscular dystrophy

Duchenne muscular dystrophy is a genetic disease that slowly destroys the muscles, including the heart muscle because one protein is missing: dystrophin. Gene therapy can correct this anomaly and recreate dystrophin production in muscle fibres. Unfortunately, because the sick fibres are abnormally fragile and permeable, the effectiveness of this therapy is low and short term. However, a solution has been found in a mouse model: the temporary stimulation of dystrophin production before gene therapy is administrated. The study showed that the muscle fibres are reinforced by the first injection and that the efficacy of gene therapy is multiplied by 10 fold for several months. Further tests are planned in animal models before moving to clinical trials.