Gene therapy may represent a promising alternative treatment of epileptic patients who are resistant to conventional anti-epileptic drugs. Among the various approaches for the application of gene therapy in the treatment of CNS disorders, recombinant adeno-associated viral (AAV) vectors have been most widely used. Preclinical studies using a selection of "therapeutic" genes injected into the rodent brain to correct the compromised balance between inhibitory and excitatory transmission in epilepsy, showed significant reduction of seizures and inhibition of epileptogenesis. In particular, transduction of neuropeptide genes, such as galanin and neuropeptide Y (NPY) in specific brain areas in experimental models of seizures resulted in significant anticonvulsant effects. Recent findings showed a long-lasting NPY over-expression in the rat hippocampus by local application of recombinant AAV vectors associated with reduced generalization of seizures, delayed kindling epileptogenesis, and strong reduction of chronic spontaneous seizures. These results establish a proof-of-principle evidence of the efficacy of gene therapy as anticonvulsant treatment. Additional investigations are required to address safety concerns and possible side effects in more detail. © 2009 The American Society for Experimental NeuroTherapeutics, Inc.

Neuropeptide Y Overexpression Using Recombinant Adenoassociated Viral Vectors / Noe, F.; Frasca, A.; Balducci, C.; Carli, M.; Sperk, G.; Ferraguti, F.; Pitkanen, A.; Bland, R.; Fitzsimons, H.; During, M.; Vezzani, A.. - In: NEUROTHERAPEUTICS. - ISSN 1933-7213. - 6:2(2009), pp. 300-306. [10.1016/j.nurt.2009.01.012]

Neuropeptide Y Overexpression Using Recombinant Adenoassociated Viral Vectors

Ferraguti F.;
2009

Abstract

Gene therapy may represent a promising alternative treatment of epileptic patients who are resistant to conventional anti-epileptic drugs. Among the various approaches for the application of gene therapy in the treatment of CNS disorders, recombinant adeno-associated viral (AAV) vectors have been most widely used. Preclinical studies using a selection of "therapeutic" genes injected into the rodent brain to correct the compromised balance between inhibitory and excitatory transmission in epilepsy, showed significant reduction of seizures and inhibition of epileptogenesis. In particular, transduction of neuropeptide genes, such as galanin and neuropeptide Y (NPY) in specific brain areas in experimental models of seizures resulted in significant anticonvulsant effects. Recent findings showed a long-lasting NPY over-expression in the rat hippocampus by local application of recombinant AAV vectors associated with reduced generalization of seizures, delayed kindling epileptogenesis, and strong reduction of chronic spontaneous seizures. These results establish a proof-of-principle evidence of the efficacy of gene therapy as anticonvulsant treatment. Additional investigations are required to address safety concerns and possible side effects in more detail. © 2009 The American Society for Experimental NeuroTherapeutics, Inc.
2009
6
2
300
306
Neuropeptide Y Overexpression Using Recombinant Adenoassociated Viral Vectors / Noe, F.; Frasca, A.; Balducci, C.; Carli, M.; Sperk, G.; Ferraguti, F.; Pitkanen, A.; Bland, R.; Fitzsimons, H.; During, M.; Vezzani, A.. - In: NEUROTHERAPEUTICS. - ISSN 1933-7213. - 6:2(2009), pp. 300-306. [10.1016/j.nurt.2009.01.012]
Noe, F.; Frasca, A.; Balducci, C.; Carli, M.; Sperk, G.; Ferraguti, F.; Pitkanen, A.; Bland, R.; Fitzsimons, H.; During, M.; Vezzani, A.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11380/1344999
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