Scientists exploring new compounds to target muscular...( Scientists have identified a promisi...),Health

Scientists have identified a promising set of new compounds in the fight against muscular dystrophy.

Using a drug-discovery technique in which molecules compete against each other for access to the target the strand of toxic RNA that causes the most common form of muscular dystrophy in adults a team at the University of Rochester Medical Center has identified several compounds that, in the laboratory, block the unwanted coupling of two molecules that is at the root of the disease.

The work was published online November 7 by the Journal of the American Chemical Society.

"This discovery gives us, for the first time, a molecule that targets the wayward RNA at the root of myotonic muscular dystrophy," said Benjamin Miller, Ph.D., the chemist who led the study. "This is a first step toward developing a drug-like molecule that perhaps could be used someday to treat the disease. This lead molecule provides a framework for moving forward."

Miller leads a team that is developing small molecules that target small strands of RNA. He notes that drugs more commonly target proteins or DNA, but that RNA offers an alluring target for some diseases.

"The drug discovery field really is wide open when it comes to RNA, which is a very difficult molecule to target," said Miller, who is an associate professor in the departments of Dermatology, Biomedical Engineering, and Biochemistry and Biophysics.

The work is the latest in a series of developments in which a Rochester team led by neurologist Charles Thornton, M.D., has shown how a genetic flaw creates the symptoms of myotonic dystrophy, which affects about 35,000 Americans.

"This is an important first step toward developing a drug treatment for myotonic dystrophy. The message from our patients is loud and clear push this forward as fast as possible," said Thornton, who is co-director of University's Neuromuscular Disease Center.

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