Article

Epilepsy gene therapy using non-integrating lentiviral delivery of an engineered potassium channel gene

2018-04-10

Abstract excerpt

Refractory focal neocortical epilepsy is a devastating disease for which there is frequently no effective treatment. Gene therapy represents a promising alternative, but treating epilepsy in this way involves irreversible changes to brain tissue, so vector design must be carefully optimized to guarantee safety without compromising efficacy. We set out to develop an epilepsy gene therapy vector optimized for clinic...

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Literature Corpus work: 9a9618a3-4cea-59bd-b998-e925e36e4686
DOI: 10.1101/298588

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