Teen Walks Independently After Treatment Targets the Genetic Root of Epilepsy
Reported byUniversity of California San Diego ↗·Sourced by Goodlede
Two children with SCN2A-related epilepsy—one aged 9, one aged 14—received personalized gene-silencing treatments delivered to spinal fluid over two years, resulting in dramatic seizure reduction and developmental gains including independent walking.
These are n-of-1 trials (one treatment per person); clinical translation to larger groups remains future research.
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