Scientists have cured mice of Parkinson's disease with gene therapy

Although there are several ways to alleviate the symptoms of Parkinson's disease, there is currently no cure or at least prevention of its development. Therefore, any methods that demonstrate efficacy in this disease are precious. Scientists at the University of California at San Diego have managed to create a therapy aimed at suppressing the activity of a single gene, which rid laboratory mice of Parkinson's disease and prevent the disease from developing for the rest of animal life. A scientific article describing the new concept has been published in the journal Nature.

Parkinson's disease develops due to neuronal death and reduced dopamine production. This leads to impairment of motor skills, balance disorders, and pain in movement. Existing treatments, such as drugs, may temporarily suppress symptoms of the disease, but they cannot fully cure the person and have serious side effects.

American researchers have developed the first experimental therapy to reverse the development of Parkinson's disease. They aimed to suppress the activity of the PBT gene and the protein of the same name, the production of which is controlled by this gene. This protein is known to prevent the formation of new neurons. During the experiment with cell cultures, they completely removed PBT from the astrocyte genome, auxiliary brain cells, as a result of which all astrocytes turned into complete neurons and were able to replace dead cells.

Scientists have attempted this technique in laboratory mice. To inhibit PBT activity, they used a harmless virus that carried artificial DNA. The results showed that astrocytes turned into neurons, and the total number of nerve cells increased by 30%. Consequently, dopamine production increased and reached healthy levels.

Observations showed that mice got rid of Parkinson's disease in three months and that she had not returned to it for the rest of her life. Next, scientists intend to conduct experiments with human cell cultures.

June 26, 2020, 12:39 p.m.

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