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Hoping for a Cure
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COLUMBIA - The 44th annual Muscular Dystrophy Association (MDA) telethon kicks off Sunday night, and many have hope that it will finally provide funds for a cure.

It may look like Dr. Dongsheng Duan is teaching a biology class, but this is no ordinary project.

"There are many, many issues," immunology and microbiology professor Dr. Duan said. "We're trying to attack one problem after another. Now we're really, really close."

Really close to a breakthrough in muscular dystrophy. Despite identifying the gene that causes it more than 30 years ago, hundreds of thousands of Americans are still waiting for some real hope of a cure.

When it comes to the most common form of muscular dystrophy, that's duchenne muscular dystrophy (DMD), it all revolves around a important protein called dystrophin. If it doesn't exist in a cell, a chain reaction starts that leads to muscle breakdown and eventually death.

"Right now we can cure a dystrophic mouse, no problem," said Dr. Duan. "Completely correct it, and the mouse will be completely normal. But if we can make it happen in a dog, that would be a big thing."

The size of the patient is the key obstacle Dr. Duan is now trying to overcome. Comparing mice to humans is a huge difference, but when it comes to body composition, comparing humans to dogs is a much smaller leap.

"So hopefully in the next 10 years down the road we'll find something to improve the patients life quality," Dr. Duan said.

Dr. Duan has already found a potential way to replace the dystrophy gene with a healthy one in mice, meaning MD disappears.

"Think about it. There are a lot of people who are suffering from this disease," Dr. Duan said. "Without the MDA funding and support, it would be impossible."

Duan also thinks an eventual neonatal test will be a reality, where doctors can find out if a baby has the malfunctioning MD gene, then replace it with a normal gene before the baby is even born.

The 44th Jerry Lewis MDA Telethon starts at 11 p.m. Sunday night and runs until 5:30 Monday evening. The local portion of the telethon will come to you from the KOMU studio in Columbia.

Reported by: Angie Bailey
Edited by: Stephanie Stouffer

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