The promise of gene therapy for the treatment of epilepsy

References

• Fisher RS, Chen DK. New routes for delivery of anti-epileptic medications. Acta Neurol. Taiwan15, 225–231 (2006).
   PubMedGoogle Scholar
• Fraser JF, Luther N, Kaplitt MG. Neurosurgical targeting, delivery, and infusion of gene therapy agents in the brain. In: Gene Therapy Of The Central Nervous System: From Bench To Bedside. Kaplitt MG, During MJ (Eds). Academic Press, NY, USA 67–75 (2006).
   Google Scholar
• Shi N, Zhang Y, Zhu C et al. Brain-specific expression of an exogenous gene after iv. administration. Proc. Natl Acad. Sci. USA98, 12754–12759 (2001).
   PubMed Web of Science ®Google Scholar
• Zhang LX, Li XL, Smith MA, Post RM, Han JS. Lipofectin-facilitated transfer of cholecystokinin gene corrects behavioral abnormalities of rats with audiogenic seizures. Neuroscience77, 15–22 (1997).
   PubMedGoogle Scholar
• Janson CG, McPhee SW, Leone P, Freese A, During MJ. Viral-based gene transfer to the mammalian CNS for functional genomic studies. Trends Neurosci.24, 706–712 (2001).
   PubMedGoogle Scholar
• Blomer U, Naldini L, Kafri T, Trono D, Verma IM, Gage FH. Highly efficient an sustained gene transfer in adult neurons with a lentivirus vector. J. Virol.71, 6641–6649 (1997).
   PubMed Web of Science ®Google Scholar
• Costantini LC, Bakowska, JC, Breakefield XO, Isacson O. Gene therapy in the CNS. Gene Ther.7, 93–109 (2000).
   PubMed Web of Science ®Google Scholar
• Mountain A. Gene therapy: the first decade. Trends Biotechnol.18, 119–128 (2000)
   PubMed Web of Science ®Google Scholar
• Ozawa CR, Ho JJ, Tsai DJ, Ho DY, Sapolsky RM. Neuroprotective potential of a viral vector system induced by a neurological insult. Proc. Natl Acad. Sci. USA97, 9270–9275 (2000).
   PubMed Web of Science ®Google Scholar
• Ho DY, McLaughlin JR, Sapolsky RM. Inducible gene expression from defective herpes simplex virus vectors using the tetracycline-responsive promoter system. Mol. Brain Res.41, 200–209 (1996).
   PubMedGoogle Scholar
• Fotaki ME, Pink JR, Mous J. Tetracycline-responsive gene expression in mouse brain after amplicon-mediated gene transfer Gene Ther.4, 901–908 (1997).
   PubMed Web of Science ®Google Scholar
• Haberman RP, Samulski RJ, McCown TJ. Attenuation of seizures and neuronal death by adeno-associated virus vector galanin expression and secretion. Nat. Med.9, 1076–1080 (2003).
   PubMed Web of Science ®Google Scholar
• Goverdhana S, Puntel M, Xiong W et al. Regulatable gene expression systems for gene therapy applications: progress and future challenges. Mol. Ther.12(2), 189–211 (2005).
   PubMed Web of Science ®Google Scholar
• Haberman R, Criswell H, Snowdy S et al. Therapeutic liabilities of in vivo viral vector tropism: adeno-associated virus vectors, NMDAR1 antisense, and focal seizure sensitivity. Mol. Ther.6, 495–500 (2002).
   PubMedGoogle Scholar
• Hommel JD, Sears RM, Georgescu D, Simmons DL, Di Leone RJ. Local gene knockdown in the brain using viral-mediated RNA interference. Nat. Med.9, 1539–1544 (2003).
   PubMed Web of Science ®Google Scholar
• Fu H, Muenzer J, Samulski RJ et al. Self-complementary adeno-associated virus serotype 2 vector: global distribution andbroad dispersion of AAV-mediated transgene expression in mouse brain. Mol. Ther.8, 911–917 (2003).
   PubMed Web of Science ®Google Scholar
• Halbert CL, Miller AD, McNamara S et al. Prevalence of neutralizing antibodies against adeno-associated virus (AAV) types 2,5, and 6 in cystic fibrosis and normal populations: implications for gene therapy using AAV vectors. Human Gene Ther.17, 440–447 (2006).
   PubMed Web of Science ®Google Scholar
• Kaplitt MG, Feigin A, Tang C et al. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson’s disease: an open label, Phase I trial. Lancet369, 2097–2105 (2007).
   PubMed Web of Science ®Google Scholar
• Yáñez-Muñoz RJ, Balaggan KS, MacNeil A. Effective gene therapy with nonintegrating lentiviral vectors. Nat. Med.12(3), 348–353 (2006).
   PubMed Web of Science ®Google Scholar
• McCarty DM, Young SM Jr, Samulski RJ. Integration of adeno-associated virus (AAV) and recombinant AAV vectors. Ann. Rev. Genet.38, 819–845 (2004).
   PubMed Web of Science ®Google Scholar
• Burger C, Gorbatyuk OS, Velardo MJ et al. Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system. Mol. Ther.10, 302–317 (2004).
   PubMed Web of Science ®Google Scholar
• Cearley CN, Wolfe JH. Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain. Mol. Ther.13, 528–537 (2006).
   PubMed Web of Science ®Google Scholar
• Boison D. Adenosine-based cell therapy approaches for pharmacoresistant epilepsies. Neurodegener. Dis.4, 28–33 (2007).
   PubMed Web of Science ®Google Scholar
• Thompson K, Anantharam V, Behrstock S et al. Conditionally immortalized cell lines, engineered to produce and release GABA, modulate the development of behavioral seizures. Exp. Neurol.161, 481–489 (2000).
   PubMed Web of Science ®Google Scholar
• Gernert M, Thompson KW, Loscher et al. Genetically engineered GABA-producing cells demonstrate anticonvulsant effects and long-term transgene expression when transplanted into the central piriform cortex of rats. Exp. Neurol.176, 183–192 (2002).
   PubMed Web of Science ®Google Scholar
• Thompson KW, Suchomelova LM. Transplants of cells engineered to produce GABA suppress spontaneous seizures. Epilepsia45, 4–12 (2004).
   PubMed Web of Science ®Google Scholar
• Richichi C, Lin EJ, Stefanin D et al. Anticonvulsant and antiepileptogenic effects mediated by adeno-associated virus vector neuropeptide Y expression in the rat hippocampus. J. Neurosci.24, 3051–3059 (2004).
   PubMed Web of Science ®Google Scholar
• Foti S, Haberman RP, Samulski RJ, McCown TJ. Adeno-associated virus-mediated expression and constitutive secretion of NPY or NPY13–36 suppresses seizure activity in vivo. Gene Ther.14(21), 1534–1536 (2007).
   PubMedGoogle Scholar
• Noe’ F, During MJ, Vezzani A. Gene therapy for epilepsy. In: Gene Therapy Of The Central Nervous System: From Bench To Bedside. Kaplitt MG, During MJ (Eds). Academic Press, NY, USA 151–163 (2006).
   Google Scholar
• Lin EJ, Richichi C, Young D et al. Recombinant AAV-mediated expression of galanin in rat hippocampus suppresses seizure development. Eur. J. Neurosci.18, 2087–2092 (2003).
   PubMedGoogle Scholar
• McCown TJ. Adeno-associated virus-mediated expression and constitutive secretion of galanin suppresses limbic seizure activity in vivo. Mol. Ther.14, 63–68 (2006).
   PubMed Web of Science ®Google Scholar
• Raol YH, Lund IV, Bandyopadhyay S et al. Enhancing GABAA receptor α1 subunit levels in hippocampal dentate gyrus inhibits epilepsy development in an animal model of temporal lobe epilepsy. J. Neurosci.26, 11342–11346 (2006).
   PubMedGoogle Scholar
• Roberts DS, Raol Y, Bandyopadhyay S et al. Erg3 stimulation of GABRA4 promoter activity as a mechanism for seizure-induced up-regulation of GABAA receptor α4 subunt expression. Proc. Natl Acad. Sci. USA102, 11894–11899 (2005).
   PubMed Web of Science ®Google Scholar
• Seki T, Matsubayashi H, Amano T et al. Adenoviral gene transfer of aspartoacylase into the tremor rat, a genetic model of epilepsy, as a trial of gene therapy for inherited epileptic disorders. Neurosci. Lett.328, 249–252 (2002).
   PubMedGoogle Scholar
• Seki T, Matsubayashi H, Amano T et al. Adenoviral gene transfer of aspartoacylase ameliorates tonic convulsions of spontaneously epileptic rats. Neurochem. Int.45, 171–178 (2004).
   PubMedGoogle Scholar
• Noe’ F, Nissinen J, Sperk G et al. NPY gene therapy in epileptic rats: inhibition of spontaneous seizures and disease-modifying effects. Epilepsia48(Suppl. 7), 19–20 (2007).
   Google Scholar
• Monahan PE, Samulski RJ. Adeno-associated virus vectors for gene therapy: more pros than cons? Mol. Med. Today6, 433–440 (2000).
   PubMedGoogle Scholar
• Ren G, Li T, Lan, JQ et al. Lentiviral RNAi-induced downregulation of adenosine kinase in human mesenchymal stem cell grafts: a novel perspective for seizure control. Exp. Neurol.208(1), 26–37 (2007).
   PubMed Web of Science ®Google Scholar
• Freese A, Kaplitt MG, O’Connor WM et al. Direct gene transfer into human epileptogenic hippocampal tissue with an adeno-associated virus vector: implications for a gene therapy approach to epilepsy. Epilepsia38, 759–766 (1997).
   PubMedGoogle Scholar
• Pitkanen A, Sutula TP. Is epilepsy a progressive disorder? Prospects for new therapeutic approaches in temporal-lobe epilepsy. Lancet Neurol.1, 173–181 (2002).
   PubMed Web of Science ®Google Scholar
• Forsyeth JR. Influence of the immune system on central nervous system gene transfer. In: Gene Therapy Of The Central Nervous System: From Bench To Bedside. Kaplitt MG, During MJ (Eds). Academic Press, NY, USA 45–52 (2006).
   Google Scholar
• Vezzani A, Granata T. Brain inflammation in epilepsy: experimental and clinical evidence. Epilepsia46, 1724–1743 (2005).
   PubMed Web of Science ®Google Scholar
• Allan SM, Tyrrell PJ, Rothwell NJ. Interleukin-1 and neuronal injury. Nat. Rev. Immunol.5, 629–640 (2005).
   PubMed Web of Science ®Google Scholar
• Tenenbaum L, Chtarto A, Lehtonen E et al. Recombinant AAV-mediated gene delivery to the central nervous system. J. Gene Med.6(Suppl. 1) S212–S222 (2004).
   PubMed Web of Science ®Google Scholar
• Lindvall O, Kokaia Z. Stem cells for the treatment of neurological disorders. Nature441, 1094–1096 (2006).
   PubMed Web of Science ®Google Scholar
• Dumas TC, Sapolsky RM. Gene therapy against neurological insults: sparing neurons versus sparing function. Trends Neurosci.24, 695–700 (2001).
   PubMed Web of Science ®Google Scholar
• Kudin AP, Debska-Vielhaber G, Vielhaber S, Elger CE, Kunz WS. The mechanism of neuroprotection by topiramate in an animal model of epilepsy. Epilepsia45, 1478–1487 (2004).
   PubMed Web of Science ®Google Scholar
• Willmore LJ. Antiepileptic drugs and neuroprotection: current status and future roles. Epilepsy Behav.7(Suppl. 3), S25–S28 (2005).
   PubMedGoogle Scholar
• Huber A, Padrun V, Deglon N, Aebischer P, Mohler H, Boison D. Grafts of adenosine-releasing cells suppress seizures in kindling epilepsy. Proc. Natl Acad. Sci. USA98, 7611–7616 (2001).
   PubMed Web of Science ®Google Scholar