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Epilepsy

Gene therapy for seizures in focal cortical dysplasia

Nature Reviews Neurology volume 20, page 63 (2024)Cite this article

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Focal cortical dysplasias (FCD) are a common cause of drug-resistant epilepsy. A new study published in Brain shows that overexpression of potassium channels through gene therapy has an antiseizure effect in a mouse model of FCD. The therapy could be suitable for clinical translation to treat epilepsy in people with FCD.

The researchers generated the FCD mouse model by in utero electroporation of frontal lobe neural progenitors with a plasmid carrying a constitutively active Ras homologue enriched in brain (RHEBCA) gene. This model was previously reported to recapitulate features of FCD type II, which is often caused by abnormal signalling of the mammalian target of rapamycin 1 pathway, downstream of RHEB.

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  1. Nature Reviews Neurology http://www.nature.com/nrneurol/

    Lisa Kiani

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  1. Lisa Kiani
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Correspondence to Lisa Kiani.

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Kiani, L. Gene therapy for seizures in focal cortical dysplasia. Nat Rev Neurol 20, 63 (2024). https://doi.org/10.1038/s41582-023-00926-8

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