Adenovirus Vector-Mediated Gene Transfer into Human Epileptogenic Brain Slices: Prospects for Gene Therapy in Epilepsy

William M. O'Connor, Beverly L. Davidson, Michael G. Kaplitt, Maureen V. Abbey, Matthew J. During, Paola Leone, David Langer, Michael J. O'Connor, Andrew Freese

Research output: Contribution to journalArticlepeer-review

19 Scopus citations


As a first step in the development of a gene therapy approach to epilepsy, we evaluated the ability of adenovirus vectors to direct the transfer into and expression of a marker gene in human brain slices obtained from patients undergoing surgery for medically intractable epilepsy. Following injection of adenovirus vectors containing the Escherichia coli lacZ gene into hippocampal and cortical brain slices, lacZ mRNA, β-galactosidase protein, and enzymatic activity were detected, confirming successful gene transfer, transcription, and translation into a functional protein. Transfected cells were predominantly glial, with some neurons expressing β-galactosidase as well. These results support the potential of adenovirus vectors to transfer genetic information into human epileptogenic brain, resulting in expression of the gene into a functional protein. These findings also have implications for the development of gene therapy approaches to certain seizure disorders. A number of potential therapeutic approaches are discussed, including the elevation of inhibitory neurotransmitter or neuropeptide levels, expression or modulation of postsynaptic receptors, and manipulation of signal transduction Systems.

Original languageEnglish (US)
Pages (from-to)167-178
Number of pages12
JournalExperimental Neurology
Issue number1
StatePublished - Nov 1997
Externally publishedYes

All Science Journal Classification (ASJC) codes

  • Neurology
  • Developmental Neuroscience


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