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What is Gene Therapy?

Gene therapy is one of the most exciting new approaches to treating drug-resistant epilepsy. But what is it? Well, as the name suggests, it’s a way of treating epilepsy by modifying a person’s genes. In fact, this can be done by: -       Inactivating a gene that is not functioning properly and may be causing the disease; or, -       Replacing that malfunctioning gene with a healthy copy; or, -       Introducing a new or modified gene to help and treat epilepsy.   The ‘treatment gene’ needs to be guided to the right place in the brain – the right cells. So, we take a harmless virus, which we call a vector. This vector will be the responsible for guiding the helpful gene into our cells. We use viruses because they are naturally very good at getting into cells, but in this case we have removed any nasty material that they might bring with them – so they are fully safe. Thus, the combination of the  helpful gene which is linked to our virus make a gene therapy construct – the treatment!   Finally, when the gene therapy is applied to the brain, the virus drives it to enter the right cells, allowing the cells to start producing the new helpful gene (or stopping the productions of the unhealthy one). When the gene is inside the cells it can be expressed forever, possibly meaning that people would not have to keep taking medications every day.   Currently up to 70 million people have epilepsy worldwide. For one third of these people, their seizures are not well-managed with currently available medications. Gene therapy is a potential solution and has shown great promise in pre-clinical lab models.   What do you think about this potential promising solution? Let us know!