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Since the cloning of the dystrophin gene in the 1980s, (2,3) the identification of its protein product, dystrophin, (4) the complex it forms in muscle, (5) and the mapping of mutations linking several MDs to dystrophin and its associated proteins, we now know a great deal about the genetic basis of these diseases. To the genome of a virus that doesn't trigger an immune response or replicate in mice or people, the researchers added the dystrophin gene, the faulty version of which causes Duchenne muscular dystrophy. After four to six weeks, the muscle will be biopsied and examined for molecular evidence of dystrophin production, representing a positive end point. |
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