What is gene therapy?

Gene therapy is a medical approach that aims to treat or prevent epilepsies known to have a genetic cause. Gene therapies enable scientists to alter a person’s genes and target areas in the brain where seizures may originate. Depending on the treatment, this can be achieved by adding or editing a single or group of genes that could cause seizures. Different gene therapies may help prevent the development of epilepsy or improve seizure control for people living with epilepsy. Clinical trials on gene therapies suggest promising for treating genetic epilepsies. However, treatment options are still in their infancy.


Gene therapy treatments for epilepsy

There are two standard gene therapy techniques for epilepsy. These include:

  • Gene addition treats genetic epilepsy by adding genetic material to a person’s cells to compensate for a missing or abnormal gene.
  • Gene editing treats genetic epilepsy by directly modifying a person’s gene (this could include gene inactivation or gene correction).


Potential benefits of gene therapy

Gene therapy for epilepsy is an evolving area with many promising signs. Benefits of gene therapy treatments so far for genetic epilepsy include:

  • Having long-lasting positives effects on epilepsy and seizure management
  • Provides additional support alongside anti-seizure medication (ASM)
  • Remove the likelihood of passing on those epilepsy genes to future offspring.


Risks of gene therapy in epilepsy

It is important to remember that gene therapy for epilepsy has some potential risks. The techniques used in gene therapy can present the following risks:

  • Gene therapy treatment could target the wrong cell
  • Increased possibility of infection
  • Cause tumours
  • Increase the risk of undesirable immune system reactions
  • Unknown long-term effects
  • Minimal evidence of efficacy


Find a clinical trial

To find clinical trials on epilepsy gene therapy:

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