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Muscular Dystrophy: Backup Gene May Help Muscles Help Themselves

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Science  04 Apr 1997:
Vol. 276, Issue 5309, pp. 35
DOI: 10.1126/science.276.5309.35

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Mammalian DNA contains redundant genes that might, in a pinch, be capable of standing in for their counterparts. Now, scientists are trying to use one of those genes to treat a currently incurable human genetic disease: Duchenne type muscular dystrophy. Each year, about 21,000 boys worldwide are born with a genetic defect that renders them incapable of making a key muscle-strengthening protein called dystrophin. Recent work in mice suggests that it may be possible to correct this defect by enticing muscle cells to make more of a very similar protein, called utrophin.