PMID- 24100249 OWN - NLM STAT- MEDLINE DCOM- 20150903 LR - 20181202 IS - 1525-5069 (Electronic) IS - 1525-5050 (Linking) VI - 38 DP - 2014 Sep TI - Gene therapy for epilepsy. PG - 125-30 LID - S1525-5050(13)00479-4 [pii] LID - 10.1016/j.yebeh.2013.09.013 [doi] AB - Gene therapy may represent an effective alternative to standard pharmacological approaches for certain forms of epilepsy. Currently, the best candidates for this therapeutic approach appear to be epilepsies characterized by a focal lesion. Gene therapy has been attempted to produce antiepileptogenic (prevention of development of epilepsy in subject at risk after having received an epileptogenic insult), antiseizure (reduction of frequency and/or severity of seizures), and disease-modifying (alteration of the natural history of the disease) effects. An example of gene therapy aimed at producing antiepileptogenic effects is a combination therapy based on the supplementation of the neurotrophic factors brain-derived neurotrophic factor (BDNF) and fibroblast growth factor 2 (FGF-2). Antiseizure effects have been obtained by increasing the strength of inhibitory signals (by supplementing specific GABAA receptor subunits or inhibitory neuropeptides like galanin or neuropeptide Y) or by reducing the strength of excitatory signals (by knocking down NMDA receptor subunits). This review summarizes the results obtained to date using gene therapy in epilepsy models and discusses the challenges and the opportunities that this approach can offer for the treatment of human epilepsies. CI - (c) 2013. FAU - Simonato, Michele AU - Simonato M AD - Department of Medical Sciences, Section of Pharmacology and Neuroscience Center, University of Ferrara, Italy; National Institute of Neuroscience, University of Ferrara, Italy; Laboratory for Technologies of Advanced Therapies (LTTA), University of Ferrara, Italy. Electronic address: michele.simonato@unife.it. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't PT - Review DEP - 20131005 PL - United States TA - Epilepsy Behav JT - Epilepsy & behavior : E&B JID - 100892858 RN - 0 (Brain-Derived Neurotrophic Factor) RN - 0 (Neuropeptide Y) RN - 103107-01-3 (Fibroblast Growth Factor 2) RN - 88813-36-9 (Galanin) SB - IM MH - Animals MH - Brain-Derived Neurotrophic Factor/genetics/*therapeutic use MH - Epilepsy/*therapy MH - Fibroblast Growth Factor 2/genetics/*therapeutic use MH - Galanin/genetics/*therapeutic use MH - Genetic Therapy/*methods MH - Humans MH - Neuropeptide Y/*therapeutic use OTO - NOTNLM OT - Brain-derived neurotrophic factor OT - Epileptogenesis OT - Galanin OT - Neuropeptide Y OT - Seizures OT - Viral vectors EDAT- 2013/10/09 06:00 MHDA- 2015/09/04 06:00 CRDT- 2013/10/09 06:00 PHST- 2013/08/30 00:00 [received] PHST- 2013/09/06 00:00 [revised] PHST- 2013/09/06 00:00 [accepted] PHST- 2013/10/09 06:00 [entrez] PHST- 2013/10/09 06:00 [pubmed] PHST- 2015/09/04 06:00 [medline] AID - S1525-5050(13)00479-4 [pii] AID - 10.1016/j.yebeh.2013.09.013 [doi] PST - ppublish SO - Epilepsy Behav. 2014 Sep;38:125-30. doi: 10.1016/j.yebeh.2013.09.013. Epub 2013 Oct 5.