Antisense compound given to mice with a disorder resembling type 1 myotonic dystrophy safely corrected molecular defects and myotonia for a year

posted on August 2, 2012 - 11:01am
An experimental treatment for type 1 myotonic muscular dystrophy (MMD1, or DM1) has corrected several aspects of the disease in an MMD1 mouse model. A year after treatment with an experimental antisense oligonucleotide dubbed ASO 445236 ended, positive effects remained apparent in the mice.

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).