Neuropeptide Y Overexpression Using Recombinant Adenoassociated Viral Vectors

Francesco Noé, Angelisa Frasca, Claudia Balducci, Mirjana Carli, Gunther Sperk, Francesco Ferraguti, Asla Pitkänen, Ross Bland, Helen Fitzsimons, Matthew During, Annamaria Vezzani

Research output: Contribution to journalArticle


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.

Original languageEnglish
Pages (from-to)300-306
Number of pages7
Issue number2
Publication statusPublished - Apr 2009


  • Adeno-associated viral vectors
  • anticonvulsant
  • gene therapy in epilepsy
  • neuropeptides
  • temporal lobe epilepsy

ASJC Scopus subject areas

  • Pharmacology (medical)
  • Clinical Neurology
  • Pharmacology

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    Noé, F., Frasca, A., Balducci, C., Carli, M., Sperk, G., Ferraguti, F., Pitkänen, A., Bland, R., Fitzsimons, H., During, M., & Vezzani, A. (2009). Neuropeptide Y Overexpression Using Recombinant Adenoassociated Viral Vectors. Neurotherapeutics, 6(2), 300-306.