09 November 2022

Gene therapy for the brain

Scientists present gene therapy for neurological and psychiatric diseases

Svetlana Maslova, Hi-tech+

The new approach is aimed exclusively at hyperactive brain cells, the excessive excitation of which provokes characteristic symptoms. Scientists have shown the first promising results in relation to epilepsy, but the potential of therapy is not limited.

Some neuropsychiatric disorders are characterized by hyperactivity of brain neurons. Gene therapy tools have the ability to correct the excitability of neurons, but so far such strategies have worked on all cells in a certain area, without distinguishing normal from hyperactive. In search of a solution, scientists from the University of California have developed a new targeted method for influencing exclusively such neurons.

Gene therapy is turned on only in hyperactive cells and is turned off when activity in a closed cycle returns to normal. To do this, certain DNA sequences of the target genes are connected to potassium channels that can reduce the excitation of cells, scientists explain. Thus, a closed cycle is created, within which it becomes possible to restore balance without affecting neighboring cells.

Using the example of a mouse model of epilepsy, it was shown that treatment reduced the frequency of seizures by 80%.

Theoretically, the new approach can be applied to the treatment of a wide variety of neurological and mental diseases. For example, in Parkinson's disease, schizophrenia, various pain and other disorders, some areas of the brain are hyperactive, so now it is possible to purposefully influence only them.

Currently, research is continuing to confirm the potential of the universality of the method, as well as its safety for humans.

Article by Qiu et al. On-demand cell-autonomous gene therapy for brain circuit disorders is published in the journal Science – VM.

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