50 million people around the world have some form of seizure. Around one-third of these people don't respond well to the treatments used to manage and prevent their seizures.
Seizures can be brought under control if the part of the brain where they occur is removed. This procedure is very dangerous.
Since epileptic seizures are caused by excessive activity of brain cells in specific parts of the brain, being able to turn them off could very well prevent them.
We were able to show in cell and animal models that it is possible to target the cells that cause epileptic seizures.
They weren't able to cause seizures in the future due to this.
There is a chance that this discovery could be used to treat Parkinson's disease and other neurological conditions caused by overproduction of dopamine.
Gene therapy alters a person's genes in order to cure a disease. There are a number of ways to do this.
Previous studies that have used gene therapy in animal models have done this by changing the virus in the lab so it's no longer harmful. The virus would be injected into the brain.
The virus would make the cells less active and prevent seizures by implanting stretches of DNA into them.
The problem with the method is that it affects all of the brain's cells, not just those that cause seizures.
The properties of the cells that take up the virally delivered DNA can be permanently altered.
It's possible to change only the brain cells that cause seizures with our new tool. We were able to take advantage of how genes are regulated.
Each of the 20,000 genes in our body has instructions for making different things. These genes are usually under the control of their neighbors. They determine how much of a particularProtein is made Depending on which promoter is active or inactive, different cells have different levels of expression.
There is a special type of promoter that only works when there is a seizure.
We were able to create a therapy that senses and turns down the excitability of neurons that cause seizures. We were able to do this by attaching activity- dependent promoter to DNA that has a calming effect on the brain.
We tested the tool in a dish and in mice that had drug-resistant seizure. In a lab-grown human, we tested this technique as well.
In each test, we were able to show that the new gene therapy technique was effective in calming down the brain cells involved in seizures.
It takes an hour or so to switch on the new gene therapy, but it is highly effective in preventing future seizures.
This can be accomplished by selecting which neurons to treat and switch off. When brain activity returns to normal, it's possible to return neuron to their original state. The promoter is ready to go if there is another seizure.
The treatment only needs to be given once, but it can last a long time.
The performance of the mice in tests of memory and other normal behavior was unaffected by the treatment.
The breakthrough could lead to the development of gene therapy for a wide range of people with drug-resistant sementans. We will need to do a number of tests to make sure that the therapy can be used with larger brains.
Gabriele Lignani is an associate professor at the University College London.
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