Isis takes on myotonic dystrophy

Researchers at the University of Rochester and Isis Pharmaceuticals Inc. have come up with an efficient way to treat symptoms of myotonic dystrophy type 1 in mice using systemically delivered antisense oligonucleotides.1 The finding has fueled an early stage development deal between Isis and Biogen Idec Inc.

Myotonic dystrophy type 1 (DM1) is a dominantly inherited condition that causes progressive muscle weakening and is the most common type of muscular dystrophy, affecting about 1 in 8,000 people. It is caused by spontaneous expansion of a trinucleotide repeat sequence in an untranslated region of the mRNA encoding dystrophia mytonica protein kinase (DMPK; DM1). The resulting transcript is abnormally large and interferes with the activity of nuclear proteins that are critical for normal muscle function.

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