A Potential Gene Therapy for Epilepsy Shows Promising Results in Laboratory Trials
Researchers from the Yong Loo Lin School of Medicine at the National University of Singapore are developing a groundbreaking treatment that could benefit epilepsy patients, especially those suffering from a rare genetic form of the disorder.
Epilepsy, characterized by recurrent seizures due to abnormal brain activity, affects millions of people worldwide. Led by Assistant Professor Huang Hua from the Department of Physiology and Neurophysiology at the School of Medicine in Singapore, the team has tested a new gene therapy targeting a specific mutation in the KCNA2 gene, which is linked to severe and recurrent seizures.
This experimental approach utilizes a therapy that targets and breaks down defective RNA, while preserving the gene’s normal function.
The treatment has demonstrated remarkable ability to restore potassium ion flow in neurons, a critical process for regulating brain activity, by addressing the underlying mutation.
Assistant Professor Huang explains: “Epilepsy often results from overactive neurons, and our treatment works to ‘repair the gate’ that controls potassium flow in the brain, helping to calm the overactive neurons and reduce seizures.”
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Although this research is still in its early stages and has been tested on cell samples, the results, published in the journal Molecular Therapy – Nucleic Acids, bring new hope to patients with genetic forms of epilepsy that do not respond to traditional treatments.
The research team is optimistic that this innovative therapy could be adapted to treat other ion channel-related disorders within the next ten to twenty years.
