Gene therapy for epilepsy

Gene therapy is being studied for some forms of epilepsy.[1] It relies on viral or non-viral vectors to deliver DNA or RNA to target brain areas where seizures arise, in order to prevent the development of epilepsy or to reduce the frequency and/or severity of seizures. Gene therapy has delivered promising results in early stage clinical trials for other neurological disorders such as Parkinson's disease,[2] raising the hope that it will become a treatment for intractable epilepsy.

  1. ^ Walker MC, Schorge S, Kullmann DM, Wykes RC, Heeroma JH, Mantoan L (September 2013). "Gene therapy in status epilepticus" (PDF). Epilepsia. 54 (Suppl 6): 43–5. doi:10.1111/epi.12275. PMID 24001071.
  2. ^ Palfi S, Gurruchaga JM, Ralph GS, Lepetit H, Lavisse S, Buttery PC, et al. (March 2014). "Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial". Lancet. 383 (9923): 1138–46. doi:10.1016/S0140-6736(13)61939-X. PMID 24412048. S2CID 4993549.