An interview with MDA research grantee Matthew Disney explores his group's findings about small molecules as a potential treatment for myotonic dystrophy
In type 1 myotonic dystrophy (MMD1, or DM1), expansions of DNA on chromosome 19 known as CTG repeats are converted to expansions in RNA called CUG repeats, which are toxic to nerve and muscles cells in a variety of ways.
Several MDA-supported research teams are targeting the toxic CUG repeats, with the goal of either blocking their interaction with other cellular substances or destroying them...
Researchers have used a 'gapmer antisense' strategy to destroy the genetic defect that causes type 1 myotonic dystrophy in cultured cells and in MMD1 mice
Researchers at Baylor College of Medicine in Houston and Isis Pharmaceuticals in Carlsbad, Calif., have announced encouraging results for their antisense-based strategy in development for the treatment of type 1 myotonic muscular dystrophy (DM1, or MMD1).