Gene therapy hope for neurological disease
Friday November 4th 2022
A new gene therapy could be a promising additional treatment for a range of neurological and psychological disorders, British scientists reported last night.
Researchers at University College London developed the therapy to target overactive neurons, which can respond poorly to drug treatments available for these diseases.
So far, the therapy has been tested on mice and the findings were reported last night in *Science*.
They found that the gene cfos promoter, in combination with the KCNA1 potassium channel gene, proved to be highly effective in calming neuronal excitability that followed induced seizure.
Researcher Dr Gabriele Lignani said: “We invented a gene therapy that switches on only in overactive cells and switches itself off if activity returns to normal.
“We harnessed the ability of certain DNA sequences to control gene expression in response to metabolic signals. By re-directing this activity-sensing mechanism to drive the production of molecules that stop brain cells from firing, we showed that epileptic seizures can be suppressed."
Fellow researcher Professor Dimitri Kullmann said: “Our findings indicate that the activity of brain cells can be normalised, and that this approach can be used to treat important neuropsychiatric diseases that do not always respond to medication.
“The gene therapy is self-regulated and can therefore be used without deciding a priori which brain cells need to be targeted.
“Importantly, it could in principle, be extended to many other disorders such as Parkinson’s disease, schizophrenia and pain disorders, where some brain circuits are overactive.”
On-demand cell-autonomous gene therapy for brain circuit disorders. *Science* 3 November 2022
Tags: Brain & Neurology | Genetics | UK News
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