Dallas Researcher Stops Progression Of Deadly Genetic Disease

Scientists in Dallas have made a big step towards stopping the most common fatal genetic disease in children.

Duchenne muscular dystrophy causes the muscles to breakdown over time.

It affects mainly boys and leads to organ failure by the time they're 30-years-old.

Scientists, for the first time, have used CRISPR gene editing to stop the progression of Duchenne muscular dystrophy in a large mammal, according to a study by UT Southwestern, that provides a strong indication that a lifesaving treatment may be in the pipeline.

Within eight weeks, the muscles in the dogs started producing the protein that the disease slowly kills.

It's called dystrophin and without it, the muscle dies over time.

"Children with DMD often die either because their heart loses the strength to pump, or their diaphragm becomes too weak to breathe," said Dr. Eric Olson, Director of UT Southwestern's Hamon Center for Regenerative Science and Medicine. 

The next step would be longer animal studies and then human trials.

"Before moving towards humans, if we are able to get there, we have to be absolutely sure that this is completely safe over the long term. That's what we are dedicated to trying to do," said Dr. Olson.

Ben Dupree, of Highland Park, is "patient zero" in Dr. Olson's research.

They used Dupree's cells and the gene editing technique called CRISPR to fix the mutation in Dupree's DNA causing his muscular dystrophy.

The breakthrough happened in a dish and the new research in animals makes Dupree excited about how his contribution to science may one day help future generations of children with Duchenne muscular dystrophy.

It may help him too.

"Just staying where I am is what I hope for the most. The promise of any amount of improvement is really exciting," said Dupree.

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