Gene therapy for epilepsy

Epilepsy and Behavior

Volumn 38, Issue , 2014, Pages 125-130

  Simonato, Michele ^a  

^a UNIVERSITY OF FERRARA   (Italy)

Author keywords

Brain derived neurotrophic factor; Epileptogenesis; Galanin; Neuropeptide Y; Seizures; Viral vectors

Indexed keywords

ANTICONVULSANT ACTIVITY; EPILEPSY; GENE TARGETING; GENE THERAPY; GENE VECTOR; HUMAN; NONHUMAN; REVIEW; ANIMAL; GENETICS; PROCEDURES;

BRAIN DERIVED NEUROTROPHIC FACTOR; FIBROBLAST GROWTH FACTOR 2; GALANIN; NEUROPEPTIDE Y;

ANIMALS; BRAIN-DERIVED NEUROTROPHIC FACTOR; EPILEPSY; FIBROBLAST GROWTH FACTOR 2; GALANIN; GENETIC THERAPY; HUMANS; NEUROPEPTIDE Y;

EID: 84910659211     PISSN: 15255050     EISSN: 15255069     Source Type: Journal    
DOI: 10.1016/j.yebeh.2013.09.013     Document Type: Review

References (39)

1. Galanopoulou A.S., Buckmaster P.S., Staley K.J., Moshe S.L., Perucca E., Engel J., et al. Identification of new epilepsy treatments: issues in preclinical methodology. Epilepsia 2012, 53:571-582.
2. Simonato M., Loscher W., Cole A.J., Dudek F.E., Engel J., Kaminski R.M., et al. Finding a better drug for epilepsy: preclinical screening strategies and experimental trial design. Epilepsia 2012, 53:1860-1867.
3. Simonato M., French J.A., Galanopoulou A.S., O'Brien T.J. Issues for new antiepilepsy drug development. Curr Opin Neurol 2013, 26:195-200.
4. Simonato M., Bennett J., Boulis N.M., Castro M.G., Fink D.J., Goins W.F., et al. Progress in gene therapy for neurological disorders. Nat Rev Neurol 2013, 9:277-291.
5. Berkovic S.F., Mulley J.C., Scheffer I.E., Petrou S. Human epilepsies: interaction of genetic and acquired factors. Trends Neurosci 2006, 29:391-397.
6. de Boer A.G., Gaillard P.J. Drug targeting to the brain. Annu Rev Pharmacol Toxicol 2007, 47:323-355.
7. Simionescu M., Popov D., Sima A. Endothelial transcytosis in health and disease. Cell Tissue Res 2009, 335:27-40.
8. Di Pasquale G., Chiorini J.A. AAV transcytosis through barrier epithelia and endothelium. Mol Ther 2006, 13:506-516.
9. Foust K.D., Nurre E., Montgomery C.L., Hernandez A., Chan C.M., Kaspar B.K. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol 2009, 27:59-65.
10. Goncalves M.A. Adeno-associated virus: from defective virus to effective vector. Virol J 2005, 2:43.
11. Matrai J., Chuah M.K., VandenDriessche T. Recent advances in lentiviral vector development and applications. Mol Ther 2010, 18:477-490.
12. Simonato M., Manservigi R., Marconi P., Glorioso J. Gene transfer into neurones for the molecular analysis of behaviour: focus on herpes simplex vectors. Trends Neurosci 2000, 23:183-190.
13. Nayak S., Herzog R.W. Progress and prospects: immune responses to viral vectors. Gene Ther 2010, 17:295-304.
14. Paradiso B., Marconi P., Zucchini S., Berto E., Binaschi A., Bozac A., et al. Localized delivery of fibroblast growth factor-2 and brain-derived neurotrophic factor reduces spontaneous seizures in an epilepsy model. Proc Natl Acad Sci U S A 2009, 106:7191-7196.
15. Richichi C., Lin E.J., Stefanin D., Colella D., Ravizza T., Grignaschi G., et al. Anticonvulsant and antiepileptogenic effects mediated by adeno-associated virus vector neuropeptide Y expression in the rat hippocampus. J Neurosci 2004, 24:3051-3059.
16. Laing J.M., Gober M.D., Golembewski E.K., Thompson S.M., Gyure K.A., Yarowsky P.J., et al. Intranasal administration of the growth-compromised HSV-2 vector deltaRR prevents kainate-induced seizures and neuronal loss in rats and mice. Mol Ther 2006, 13:870-881.
17. Gray S.J., Blake B.L., Criswell H.E., Nicolson S.C., Samulski R.J., McCown T.J., et al. Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB). Mol Ther 2010, 18:570-578.
18. Dudek F.E. Commentary: a skeptical view of experimental gene therapy to block epileptogenesis. Neurotherapeutics 2009, 6:319-322.
19. Paradiso B., Zucchini S., Su T., Bovolenta R., Berto E., Marconi P., et al. Localized overexpression of FGF-2 and BDNF in hippocampus reduces mossy fiber sprouting and spontaneous seizures up to 4weeks after pilocarpine-induced status epilepticus. Epilepsia 2011, 52:572-578.
20. Bovolenta R., Zucchini S., Paradiso B., Rodi D., Merigo F., Navarro Mora G., et al. Hippocampal FGF-2 and BDNF overexpression attenuates epileptogenesis-associated neuroinflammation and reduces spontaneous recurrent seizures. J Neuroinflammation 2010, 7:81.
21. Simonato M., Tongiorgi E., Kokaia M. Angels and demons: neurotrophic factors and epilepsy. Trends Pharmacol Sci 2006, 27:631-638.
22. Simonato M., Zucchini S. Are the neurotrophic factors a suitable therapeutic target for the prevention of epileptogenesis?. Epilepsia 2010, 51(Suppl. 3):48-51.
23. Thoenen H., Sendtner M. Neurotrophins: from enthusiastic expectations through sobering experiences to rational therapeutic approaches. Nat Neurosci 2002, 5:1046-1050. [Suppl.].
24. Brooks-Kayal A.R., Shumate M.D., Jin H., Rikhter T.Y., Coulter D.A. Selective changes in single cell GABA(A) receptor subunit expression and function in temporal lobe epilepsy. Nat Med 1998, 4:1166-1172.
25. Raol Y.H., Lund I.V., Bandyopadhyay S., Zhang G., Roberts D.S., Wolfe J.H., et al. Enhancing GABA(A) receptor alpha 1 subunit levels in hippocampal dentate gyrus inhibits epilepsy development in an animal model of temporal lobe epilepsy. J Neurosci 2006, 26:11342-11346.
26. Haberman R., Criswell H., Snowdy S., Ming Z., Breese G., Samulski R., et al. Therapeutic liabilities of in vivo viral vector tropism: adeno-associated virus vectors, NMDAR1 antisense, and focal seizure sensitivity. Mol Ther 2002, 6:495-500.
27. Wykes R.C., Heeroma J.H., Mantoan L., Zheng K., MacDonald D.C., Deisseroth K., et al. Optogenetic and potassium channel gene therapy in a rodent model of focal neocortical epilepsy. Sci Transl Med 2012, 4. [161ra152].
28. Kanter-Schlifke I., Georgievska B., Kirik D., Kokaia M. Seizure suppression by GDNF gene therapy in animal models of epilepsy. Mol Ther 2007, 15:1106-1113.
29. Theofilas P., Brar S., Stewart K.A., Shen H.Y., Sandau U.S., Poulsen D., et al. Adenosine kinase as a target for therapeutic antisense strategies in epilepsy. Epilepsia 2011, 52:589-601.
30. Lerner J.T., Sankar R., Mazarati A.M. Galanin and epilepsy. Cell Mol Life Sci 2008, 65:1864-1871.
31. Haberman R.P., Samulski R.J., McCown T.J. Attenuation of seizures and neuronal death by adeno-associated virus vector galanin expression and secretion. Nat Med 2003, 9:1076-1080.
32. Lin E.J., Richichi C., Young D., Baer K., Vezzani A., During M.J. Recombinant AAV-mediated expression of galanin in rat hippocampus suppresses seizure development. Eur J Neurosci 2003, 18:2087-2092.
33. McCown T.J. Adeno-associated virus-mediated expression and constitutive secretion of galanin suppresses limbic seizure activity in vivo. Mol Ther 2006, 14:63-68.
34. Noe F., Frasca A., Balducci C., Carli M., Sperk G., Ferraguti F., et al. Neuropeptide Y overexpression using recombinant adeno-associated viral vectors. Neurotherapeutics 2009, 6:300-306.
35. Foti S., Haberman R.P., Samulski R.J., McCown T.J. Adeno-associated virus-mediated expression and constitutive secretion of NPY or NPY13-36 suppresses seizure activity in vivo. Gene Ther 2007, 14:1534-1536.
36. Woldbye D.P., Angehagen M., Gotzsche C.R., Elbrond-Bek H., Sorensen A.T., Christiansen S.H., et al. Adeno-associated viral vector-induced overexpression of neuropeptide Y Y2 receptors in the hippocampus suppresses seizures. Brain 2010, 133:2778-2788.
37. Noe F., Pool A.H., Nissinen J., Gobbi M., Bland R., Rizzi M., et al. Neuropeptide Y gene therapy decreases chronic spontaneous seizures in a rat model of temporal lobe epilepsy. Brain 2008, 131:1506-1515.
38. Sorensen A.T., Nikitidou L., Ledri M., Lin E.J., During M.J., Kanter-Schlifke I., et al. Hippocampal NPY gene transfer attenuates seizures without affecting epilepsy-induced impairment of LTP. Exp Neurol 2009, 215:328-333.
39. Noe F., Vaghi V., Balducci C., Fitzsimons H., Bland R., Zardoni D., et al. Anticonvulsant effects and behavioural outcomes of rAAV serotype 1 vector-mediated neuropeptide Y overexpression in rat hippocampus. Gene Ther 2010, 17:643-652.