2017 ERUK Fellow Dr Gabriele Lignani, has been awarded a New Investigator Research Grant, worth nearly £700,000 by the Medical Research Council (MRC), to investigate the potential use of gene therapy in Dravet syndrome. This research project will build on Dr Lignani’s ongoing fellowship research – GeneLoop, which is currently investigating the potential of gene therapy as a treatment for acquired intractable epilepsy.
Dravet Syndrome is a severe and extremely rare but catastrophic neurological disorder affecting young children (approximately 1 in 19,000 people). 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 some of the most commonly prescribed and effective antiepileptic drugs may even worsen Dravet syndrome.
Dr Lignani said: “This research will be useful not only to understand if a permanent general treatment for Dravet syndrome is achievable, but will also give insight into the potential of the techniques to cure other neurological conditions caused by similar mutations in different genes.”
He continued “This research will be useful not only to understand if a permanent general treatment for Dravet syndrome is achievable, but will also give insight into the potential of the techniques to cure other neurological conditions caused by similar mutations in different genes.”
Dr Lignani’s ERUK Fellowship officially began in 2018 but is already showing promising results. His team’s work thus far has focused on developing and testing their gene therapy tools, and they have found that this tool is able to subdue neurons during epileptic activity. The following stages of the fellowship will involve trialing these tools in models of acquired intractable epilepsy.