Technology

Personalized gene therapy reduces seizures in two boys with severe epilepsy

A custom antisense oligonucleotide approach targeting SCN2A mutations shows early promise for rare developmental epileptic encephalopathy.

Talivio News · Global 24 July 2026, 16:10 1 min read

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Young boy sitting in a therapy room during a session, focused on an activity.

A personalized gene therapy has reduced seizures in two boys suffering from a rare and severe form of epilepsy, according to a study published in Nature Medicine. The treatment uses individualized antisense oligonucleotides to target faulty mRNA caused by specific mutations in the SCN2A gene.

The therapy is designed for allele-specific faulty mRNA in patients with SCN2A mutations, a genetic cause of developmental epileptic encephalopathy. The antisense oligonucleotides are customized to each patient's unique mutation, aiming to correct the underlying genetic defect.

While the results are early and based on a small number of patients, they represent a step toward individualized treatments.

Elian Kastrup

Technology Correspondent · Talivio News

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Sources

nature.com — Personalized gene therapy relieves severe epilepsy in two boys (opens in a new window) accessed 24 Jul 2026
Hackaday — Targeting Allele-Specific Faulty MRNA In SCNA2 Mutation Patients (opens in a new window) accessed 24 Jul 2026

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