JUPITER, FL — People diagnosed with myotonic dystrophy type 1 have difficulty unclenching muscles due to a type of genetic defect that generates toxic material within their cells. There is currently no treatment. In a new report published in the Proceedings of the National Academy of Sciences, a group at Scripps Research in Florida says they have made a potential drug that targets a disease-causing RNA at the root of the disease. In mouse and cellular models of myotonic dystrophy type 1, it improved the muscle defects with no apparent side-effects.
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