Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2014 May;10(5):300-4.
doi: 10.1038/nrneurol.2014.43. Epub 2014 Mar 18.

Gene therapy in epilepsy-is it time for clinical trials?

Dimitri M Kullmann  1 , Stephanie Schorge  1 , Matthew C Walker  1 , Robert C Wykes  1
Affiliations
Review

Gene therapy in epilepsy-is it time for clinical trials?

Dimitri M Kullmann et al. Nat Rev Neurol. 2014 May.

Abstract

Epilepsy represents a major burden to society, not least because approximately 25% of patients do not respond satisfactorily to antiepileptic medication, and only a minority with pharmacoresistant epilepsy are eligible for potentially curative surgery. Several studies have explored gene therapy as a treatment strategy. The translation of scientific breakthroughs into the clinic faces several challenges, including the validation of experimental models of human pharmacoresistant epilepsy, establishment of sensitive and specific measures of therapeutic efficacy, and evaluation of the long-term safety of gene therapy. On the basis of successful reports of gene therapy in experimental models of epilepsy, a roadmap toward clinical trials is proposed.

PubMed Disclaimer

References

    1. Brain. 2008 Jun;131(Pt 6):1506-15 - PubMed
    1. Epilepsia. 2011 Mar;52(3):572-8 - PubMed
    1. J Neurol Neurosurg Psychiatry. 1977 Apr;40(4):404-14 - PubMed
    1. Epilepsia. 2008 Jul;49(7):1230-8 - PubMed
    1. Hum Gene Ther. 2005 Jun;16(6):741-51 - PubMed

Publication types