Recent advances and current status of gene therapy for epilepsy

Fiest KM, Sauro KM, Wiebe S, Patten SB, Kwon CS, Dykeman J, et al. Prevalence and incidence of epilepsy: a systematic review and meta-analysis of international studies. Neurology. 2017;88:296–303.

Article  PubMed  PubMed Central  Google Scholar 

Keezer MR, Sisodiya SM, Sander JW. Comorbidities of epilepsy: current concepts and future perspectives. Lancet Neurol. 2016;15:106–15.

Article  PubMed  Google Scholar 

Schulze-Bonhage A. A 2017 review of pharmacotherapy for treating focal epilepsy: where are we now and how will treatment develop? Expert Opin Pharmacother. 2017;18:1845–53.

Article  PubMed  Google Scholar 

Chen Z, Brodie MJ, Liew D, Kwan P. Treatment outcomes in patients with newly diagnosed epilepsy treated with established and new antiepileptic drugs: a 30-year longitudinal cohort study. JAMA Neurol. 2018;75:279–86.

Article  PubMed  Google Scholar 

Elmslie F, Gardiner M. Genetics of the epilepsies. Curr Opin Neurol. 1995;8:126–9.

Article  PubMed  Google Scholar 

Perucca P, Perucca E. Identifying mutations in epilepsy genes: impact on treatment selection. Epilepsy Res. 2019;152:18–30.

Article  PubMed  Google Scholar 

Symonds JD, Zuberi SM. Genetics update: monogenetics, polygene disorders and the quest for modifying genes. Neuropharmacology. 2018;132:3–19.

Article  PubMed  Google Scholar 

Street JS, Qiu Y, Lignani G. Are genetic therapies for epilepsy ready for the clinic? Epilepsy Curr. 2023;23:245–50.

Article  PubMed  PubMed Central  Google Scholar 

Lieb A, Qiu Y, Dixon CL, Heller JP, Walker MC, Schorge S, et al. Biochemical autoregulatory gene therapy for focal epilepsy. Nat Med. 2018;24:1324–9.

Article  PubMed  PubMed Central  Google Scholar 

Wang X, Tian X, Yang Y, Lu X, Li Y, Ma Y, et al. POSH participates in epileptogenesis by increasing the surface expression of the NMDA receptor: a promising therapeutic target for epilepsy. Expert Opin Ther Targets. 2017;21:1083–94.

Article  PubMed  Google Scholar 

Chen L, Gao B, Fang M, Li J, Mi X, Xu X, et al. Lentiviral vector-induced overexpression of RGMa in the hippocampus suppresses seizures and mossy fiber sprouting. Mol Neurobiol. 2017;54:1379–91.

Article  PubMed  Google Scholar 

Kong Q, Takahashi K, Schulte D, Stouffer N, Lin Y, Lin CL. Increased glial glutamate transporter EAAT2 expression reduces epileptogenic processes following pilocarpine-induced status epilepticus. Neurobiol Dis. 2012;47:145–54.

Article  PubMed  PubMed Central  Google Scholar 

Green JL, Dos Santos WF, Fontana ACK. Role of glutamate excitotoxicity and glutamate transporter EAAT2 in epilepsy: opportunities for novel therapeutics development. Biochem Pharmacol. 2021;193:114786.

Article  PubMed  PubMed Central  Google Scholar 

Niibori Y, Lee SJ, Minassian BA, Hampson DR. Sexually divergent mortality and partial phenotypic rescue after gene therapy in a mouse model of Dravet syndrome. Hum Gene Ther. 2020;31:339–51.

Article  PubMed  PubMed Central  Google Scholar 

Zhang L, Sun W, Xu L, Wang Y, Zhu G, Wu X, et al. The anticonvulsant and neuroprotective effects of kir2.3 activation in PTZ-induced seizures and the kainic acid model of TLE. Epilepsy Res. 2019;156:106167.

Article  PubMed  Google Scholar 

Wykes RC, Heeroma JH, Mantoan L, Zheng K, MacDonald DC, Deisseroth K, et al. Optogenetic and potassium channel gene therapy in a rodent model of focal neocortical epilepsy. Sci Transl Med. 2012;4:161.

Article  Google Scholar 

Snowball A, Chabrol E, Wykes RC, Shekh-Ahmad T, Cornford JH, Lieb A, et al. Epilepsy gene therapy using an engineered potassium channel. J Neurosci. 2019;39:3159–69.

Article  PubMed  PubMed Central  Google Scholar 

Lin EJ, Young D, Baer K, Herzog H, During MJ. Differential actions of NPY on seizure modulation via Y1 and Y2 receptors: evidence from receptor knockout mice. Epilepsia. 2006;47:773–80.

Article  PubMed  Google Scholar 

Gotzsche CR, Nikitidou L, Sorensen AT, Olesen MV, Sorensen G, Christiansen SH, et al. Combined gene overexpression of neuropeptide Y and its receptor Y5 in the hippocampus suppresses seizures. Neurobiol Dis. 2012;45:288–96.

Article  PubMed  Google Scholar 

Noe F, Nissinen J, Pitkanen A, Gobbi M, Sperk G, During M, et al. Gene therapy in epilepsy: the focus on NPY. Peptides. 2007;28:377–83.

Article  PubMed  Google Scholar 

Richichi C, Lin EJ, 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;24:3051–9.

Article  PubMed  PubMed Central  Google Scholar 

Powell KL, Fitzgerald X, Shallue C, Jovanovska V, Klugmann M, Von Jonquieres G, et al. Gene therapy mediated seizure suppression in genetic generalised epilepsy: neuropeptide Y overexpression in a rat model. Neurobiol Dis. 2018;113:23–32.

Article  PubMed  Google Scholar 

Ledri LN, Melin E, Christiansen SH, Gotzsche CR, Cifra A, Woldbye DP, et al. Translational approach for gene therapy in epilepsy: model system and unilateral overexpression of neuropeptide Y and Y2 receptors. Neurobiol Dis. 2016;86:52–61.

Article  PubMed  Google Scholar 

Melin E, Andersson M, Gotzsche CR, Wickham J, Huang Y, Szczygiel JA, et al. Combinatorial gene therapy for epilepsy: gene sequence positioning and AAV serotype influence expression and inhibitory effect on seizures. Gene Ther. 2023;30:649–58.

Article  PubMed  PubMed Central  Google Scholar 

Drew L. Gene therapy targets epilepsy. Nature. 2018;564:S10–1.

Article  PubMed  Google Scholar 

Szczygiel JA, Danielsen KI, Melin E, Rosenkranz SH, Pankratova S, Ericsson A, et al. Gene therapy vector encoding neuropeptide Y and its receptor Y2 for future treatment of rpilepsy: preclinical data in rats. Front Mol Neurosci. 2020;13:232.

Article  PubMed  PubMed Central  Google Scholar 

Patricio MI, Barnard AR, Green AL, During MJ, Sen A, MacLaren RE. A clinical-grade gene therapy vector for pharmacoresistant epilepsy successfully overexpresses NPY in a human neuronal cell line. Seizure. 2018;55:25–9.

Article  PubMed  Google Scholar 

Freimann K, Kurrikoff K, Langel U. Galanin receptors as a potential target for neurological disease. Expert Opin Ther Targets. 2015;19:1665–76.

Article  PubMed  Google Scholar 

McCown TJ. Adeno-associated virus-mediated expression and constitutive secretion of galanin suppresses limbic seizure activity in vivo. Mol Ther. 2006;14:63–8.

Article  PubMed  Google Scholar 

Ledri M, Sorensen AT, Madsen MG, Christiansen SH, Ledri LN, Cifra A, et al. Differential effect of neuropeptides on excitatory synaptic transmission in human epileptic hippocampus. J Neurosci. 2015;35:9622–31.

Article  PubMed  PubMed Central  Google Scholar 

Guipponi M, Chentouf A, Webling KE, Freimann K, Crespel A, Nobile C, et al. Galanin pathogenic mutations in temporal lobe epilepsy. Hum Mol Genet. 2015;24:3082–91.

Article  PubMed  Google Scholar 

Collard R, Aziz MC, Rapp K, Cutshall C, Duyvesteyn E, Metcalf CS. Galanin analogs prevent mortality from seizure-induced respiratory arrest in mice. Front Neural Circuits. 2022;16:901334.

Article  PubMed  PubMed Central  Google Scholar 

Xu X, Guo F, Cai X, Yang J, Zhao J, Min D, et al. Aberrant changes of somatostatin and neuropeptide Y in brain of a genetic rat model for epilepsy: tremor rat. Acta Neurobiol Exp (Wars). 2016;76:165–75.

Article  PubMed  Google Scholar 

Yekhlef L, Breschi GL, Lagostena L, Russo G, Taverna S. Selective activation of parvalbumin- or somatostatin-expressing interneurons triggers epileptic seizurelike activity in mouse medial entorhinal cortex. J Neurophysiol. 2015;113:1616–30.

留言 (0)

沒有登入
gif