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Researchers in China have reported the first clinical results using a chemogenetic approach to treat epilepsy, treating three patients with a novel gene therapy combined with a drug to reduce seizure activity. The therapy involves introducing genes into brain cells that make them responsive to a specific drug, which then dampens excessive neuronal activity associated with seizures. This represents the first human application of chemogenetics, a technique previously used only in laboratory research.
Why it matters
This approach could offer a new treatment option for patients with drug-resistant epilepsy who cannot be helped by conventional medications. If proven effective in larger trials, chemogenetics could potentially be adapted to treat other neurological conditions involving abnormal brain cell activity.
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In first clinical result using “chemogenetic” strategy, a team in China treated three patients to control epilepsy
Source: Novel gene therapy lets drug dampen brain cell activity, reducing seizures