Dravet Syndrome

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Dravet Syndrome is a severe catastrophic neurological disorder affecting young children. Every day 3 or 4 children are diagnosed with Dravet syndrome worldwide, with symptoms including epilepsy, autism, movement disorders and sleep disturbances. To date, the majority of therapies are ineffective or poorly tolerated. Sodium channel drugs which are otherwise effective in many forms of epilepsy may even worsen Dravet syndrome. In the lab we are aiming for a therapy that will restore the normal functioning of the brain in Dravet, using CRISPR and other innovative approaches, but also to study the pathophysiology of this devastating childhood disease.