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


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.

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Figure 1: A patient-centred approach to gene therapy for epilepsy.


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Work in the authors' laboratories is supported by the Wellcome Trust, Medical Research Council, European Research Council and the Royal Society.

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All authors researched the data for the article, provided substantial contributions to discussions of its content, wrote the article and undertook review and/or editing of the manuscript before submission.

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Correspondence to Dimitri M. Kullmann.

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The authors declare no competing financial interests.

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Kullmann, D., Schorge, S., Walker, M. et al. Gene therapy in epilepsy—is it time for clinical trials?. Nat Rev Neurol 10, 300–304 (2014).

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