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
posted on February 29, 2012 - 9:58am
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).
Scientists have developed synthetic compounds that reduce the harmful effects of MMD1-associated expansions of RNA in cellular models of the disease
posted on February 23, 2012 - 3:45pm
Editor's note 3/15/12: This story was updated to reflect the availability of a podcast with Matthew Disney.
Small, laboratory-designed molecules can make a big difference in cells carrying the genetic defect that causes type 1 myotonic dystrophy (DM1, or MMD1), researchers have found.