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UVA gene therapy treatment for severe form of epilepsy reaches clinical trials

UVA Gene Therapy
Published: Jan. 18, 2022 at 4:40 PM EST
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CHARLOTTESVILLE, Va. (WVIR) - Recent research from the University of Virginia School of Medicine could soon help people with epilepsy.

Researchers are working on developing new gene therapy to fight Dravet Syndrome, a kind of severe epilepsy. They say this work is especially important, because it targets the gene mutation that causes seizures, fixing the root of the issue.

Doctor Eric Wengert is a former UVA graduate student who worked on this gene therapy development. He says the treatments have so far been successful in mice.

“The mice have the exact same genetic change that the patients have and the mice have seizures, and sometimes those seizures lead to death. When we measured seizures in the the mice treated with new gene therapy only one had seizures and then died,” Wengert said.

The therapy is now entering clinical trials to research how humans handle it.

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