Teen Walks Independently After Treatment Targets the Genetic Root of Epilepsy

UC San Diego and Rady Children’s researchers used personalized allele-selective antisense oligonucleotides to treat two children with SCN2A-related developmental epileptic encephalopathy.

Over two years, both children had fewer seizures, needed less medication, and showed developmental gains with few side effects.

The older child later began walking independently, highlighting the potential of precision gene therapy to treat epilepsy at its root cause.