Researchers have revealed an atomic-level view of a genetic defect that causes a form of muscular dystrophy myotonic dystrophy type 2. The scientists from The Scripps Research Institute have used this information to design drug candidates with potential to counter those defects and reverse the disease. 'This the first time the structure of the RNA defect that causes this disease has been determined' lead author Matthew Disney said. 'Based on these results we designed compounds that even in small amounts significantly improve disease-associated defects in treated cells.' Myotonic dystrophy type 2 is a relatively rare form of muscular dystrophy that
