A Cure for Clara: Gene therapy developed at Auburn University set for human trials

Clara suffers from rare, fatal genetic disorder

The race to find a cure for a rare genetic disease has become a Hoover family's mission as they try to save their little girl. "A Cure for Clara," may come from of all places Auburn University's College of Veterinary Medicine.

Everything appeared normal when Baby Clara came into the world. By 14 months though, she was lagging behind in development. "Our first red flag, she wasn't walking," explains her mom Jenny Bragg. Then the heartbreaking diagnosis came last August. Clara had GM1 gangliosidosis which is an inherited disorder. It destroys nerve cells.

"She was terminal; they said there was nothing they could do for her and we should go home and enjoy our time with her," recalls Bragg with tears in her eyes. She and her husband scoured the internet looking for something, any hope.

That lead them to Auburn University and groundbreaking research at the College of Veterinary Medicine. GM1 had been cured in cats and the researchers were preparing for human clinical trials. The gene therapy involves a single IV injection.

A research cat named Cinnamon who was treated is now seven years old. Others have also been cured. "They could live a normal life span. Showing this treatment works in animals is the first step to see if it's applicable to humans," explains Auburn Researcher and Professor Doug Martin, Ph.D.

The remarkable results hold promise for curing other fatal diseases. "If we can find the gene that causes Huntington's disease, Lou Gehrig's disease, the same basic technique and approach can be used," says Martin.

Human trials are set for six children including Clara if she stays healthy in November at the National Institutes of Health in Bethesda, Maryland. "I do have apprehension . on the other hand it's our only shot saving her life," says Jenny Bragg.

To make sure those human trials happen another $400,000 needs to be raised. A special fundraiser is set for Saturday, April 8th at the Redmont Hotel: Clara's Birthday Bash.

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All the proceeds go to the Cure GM1 Foundation.

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