Scientists Reverse Muscular Dystrop...(g adults. It is caused when a section of...),Health

There is no cure for the disease nor are there many effective treatments. In fact, scientists are only now beginning to understand the mechanisms behind muscular dystrophy.

Several years ago, this same group of researchers showed that a genetic flaw leads to the overproduction of a certain type of messenger RNA (mRNA) in cells, which affects muscle control and other cellular functions.

The original defects in the DNA translate into expanded repeats in the RNA which then bind up various cellular proteins. These include one called "muscleblind," thought to be a key factor in myotonic dystrophy.

"Probably the main thing [the RNA] does is to act like a sponge and sop up some of the important proteins," said study senior author Dr. Charles Thornton, professor of neurology at the University of Rochester Medical Center and co-director of the University of Rochester Medical Center's Wellstone Muscular Dystrophy Cooperative Research Center.

This is the first time RNA has emerged as a culprit in a disease process, he said.

"We don't have to replace a bad gene. All we have to do is to keep this toxic RNA and these proteins from sticking to one another," continued Thornton. "In theory, if you could make that happen might you might actually make this disease go in reverse."

And the hypothesis panned out.

An "antisense morpholino oligonucleotide," a synthetic molecule, succeeded in pulling apart the RNA and the muscleblind proteins.

Using this molecule, "we neutralized a toxic RNA so it released the protein stuck on it," Thornton said.

A major challenge now will be to figure out a way to deliver the therapy to
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