Promising gene therapy for Parkinson's disease has been developed

Loss of dopamine neurons in a substania nigra is a feature of Parkinson's disease. Most treatments for this disease aim to block this process or protect vulnerable neural circuits, but such approaches do not reverse neurodegeneration. A team of researchers from the University of San Diego (USA) offers an alternative way: to treat Parkinson's disease by replacing the lost dopamine-producing neurons to restore the broken connection - by special gene therapy. A scientific article about this is published in the journal Nature.

In the course of experiments with laboratory mice, scientists managed to block the activity of the PBT gene and thus disrupt the synthesis of RNA-binding protein. This allowed astrocytes, the cells of glia (brain auxiliary nervous tissue), to turn into functional neurons. 

To silence the PBT gene, specialists injected into the cells a special virus carrying a piece of DNA called an anti-sensory oligonucleotide. Such changing contributed to the fact that the PBT gene stopped coding the corresponding protein, and this allowed new neurons to form.

Thanks to gene therapy, the number of neurons in the brain of mice has increased by 30% and dopamine levels have returned to normal. Animals have returned to motor ability, motor function. They got rid of the symptoms for the rest of their lives. By comparison, there was no such improvement in the control group.

According to the authors, new gene therapy could be a potential treatment for Parkinson's disease in humans. However, additional testing on mouse models is required first before proceeding to test on human cells.

July 23, 2020, 11:01 a.m.

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