Clemson researchers work to change DNA, prevent disease - FOX Carolina 21

Clemson researchers work to change DNA, prevent disease

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CLEMSON, SC (FOX Carolina) - Stopping disease before it starts, changing DNA, finding treatments and reversing debilitating disorders, are all types of personalized medicine that may arrive in the not-too distant future. Researchers at Clemson University are on the front lines of making it happen.

The work is in beginning stages, using Clemson's super computer, The Palmetto Cluster, for complex calculations, but doctors and patients expect the work to change their world.

It's a change that Connor Raymond's family can't see come soon enough.

Even as a baby, now-six-year-old Connor's parents knew something wasn't quite right. His mom, Katia Luedtke, remembers that he was missing milestones that typical babies made. She said that by six-months-old, Connor couldn't sit up, roll over, or hold his head up.

After years of testing, Connor was diagnosed with Snyder-Robinson Syndrome, a genetic disorder that affects the body and intellectual capacity.

As one of just 821 in the world with Snyder-Robinson, Connor's family was astonished to find that Dr. Emil Alexov, at Clemson University's Department of Physics, was studying their son's disorder.

Alexov and his team of post-doctorate fellows use computer modeling to analyze individual DNA molecules in people with many different mental disorders. He said they're trying to figure out how small differences in DNA affects human health.

Since each molecule is made of hundreds of thousands of atoms, the team needs The Palmetto Cluster to make millions of computations. On a regular computer, that could take years, but even on the super computer, one calculation could take a couple weeks.

Alexov said the most important part is to figure out how the molecule acts when it's healthy and when it's not. That, he said, will bring them closer to developing treatment.

Eventually, they want to physically fix a genetic difference.

“In this system, we found the variant causes the protein to be less stable, so we're trying to figure out how we can reverse this effect to rescue the natural stability of the protein,” said fellow, Tugba Kucukkai.

While Alexov's team works in a computer lab, they corroborate with geneticists, and scientists like Dr. Charles Schwartz, at the Greenwood Genetics Center, who work directly with patients.

Since Schwartz' office discovered the gene mutation that causes Snyder-Robinson, he now knows the Raymonds in Virginia.

Now, Connor is on seizure medication, and his family anticipates bone deterioration over the years.

“We may be able to treat those situations, the seizures or the osteoporosis or the scoliosis, and not impact the cognitive function, because the cognitive function may already be set in place and it may be difficult to reverse that. Whereas the seizures, it may be able to mitigate that,” said Schwartz.

To the Raymonds, that would be a huge improvement.

“As he gets older, things will become more difficult. He won't remain a cute little boy. He'll be very fragile, and with intellectual disability, there's no telling if he'll be ever be able to hold a job or have a relationship,” said Luedtke.

Schwartz thinks it could take years to translate Dr. Alexov's computer modeling into actual treatment with lab-testing, before human testing. That's why Alexov looks at drugs that are already FDA approved, to see if they could be used in different capacities. That gives the Raymonds hope for a speedier treatment.

Talk of manipulating DNA leads to ethical questions, like, whose decision will it be, and to use which kind of treatment?

Dr. Howard Bean, with Mary Black Physician's Group, said that he works with children and adolescents with ADD who don't always say they want to be, “normal,” and “cured” of their disorder.

Bean does think that working on molecules and changing someone's genetic makeup is the way of the future.

“I think that if we sit back and wait on our legal system or ethics committees and such to decide on these things, much of it will never happen. It will be too much time talking and not enough time doing,” said Bean.

The "doing," is something Connor Raymond's family is anxious to see come true.

At this point, personalized medicine usually means comparing DNA differences, against known diseases. Doctors prescribe preventative healthy habits, or even drugs to ease symptoms, but manipulating molecules as the folks at Clemson are working on could mean a whole new frontier of finding cures.

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