Exploring gene therapy with Oxford University
Lead researcher: Professor Matthew Wood
Partner: University of Oxford | Great Ormond Street Hospital Children's Charity
Start date: 2021
This research aims to find a new way to treat Dravet Syndrome by using a special editing technique, called RNA editing, which changes the instructions produced by the SCN1A gene. This will hopefully mean that this gene doesn’t lead to the faulty functioning of an important type of protein in the brain that can lead to seizures and other Dravet Syndrome symptoms.
The research team will first test the editing techniques on cells in a dish and then move on to investigating the effects on mice.
We’re a few years away from knowing if this could make a difference to families affected by Dravet Syndrome. But exploratory studies like this project are very important in contributing to our knowledge of the underlying causes of Dravet Syndrome and how to address these.
Some of the editing techniques have already been used to treat conditions, like spinal muscular atrophy.