BioCentury
ARTICLE | Tools & Techniques

Targeting toxic RNA

August 6, 2009 7:00 AM UTC

Researchers at the University of Rochester have used an antisense molecule to reverse muscular defects in mice with myotonic dystrophy type 1.1 The approach represents a new way to attack the most common form of adult muscular dystrophy and may be applicable to other diseases that are also characterized by expanded RNA repeats.

Myotonic dystrophy type 1 (DM1) is an RNA-mediated, dominantly inherited disease caused by long triplet repeat regions of CUG in the noncoding region of the RNA that is translated into dystrophia myotonica protein kinase (DMPK). Whereas healthy people have about 5-30 copies of the triplet repeat, DM1 patients have hundreds to thousands of copies...