Gene therapy cured mice of Alzheimer's disease

RIA News

Molecular biologists have created and successfully tested the first retroviral therapy for the treatment of Alzheimer's disease, relieving and protecting mice from the consequences of its development, according to an article published in the journal PNAS (Katsouri et al., PPARy-coactivator-1α gene transfer reduces neuronal loss and amyloid-β generation by reducing β-secretase in an Alzheimer’s disease model).

"Although it is still very early to talk about it, the results of our experiments suggest that gene therapy can be used to treat Alzheimer's disease. To do this, we will have to overcome a lot of obstacles, and so far we cannot introduce such gene therapy in any other way than by direct injection into the brain," said Magdalena Sastre from Imperial College London.

Images from Alzheimer's disease press release could be treated with gene therapy, suggests animal study. Amyloid plaques are shown in green. On the left – before gene therapy, on the right – after it – VM.

It is believed that Alzheimer's disease is caused by the accumulation of a pathogenic substance inside neurons, the beta-amyloid protein. It is formed from the "scraps" of the APP protein, which is involved in the processes of repairing damaged neurons and forming connections between them. Violations in the processing of molecules of this protein lead to the appearance of beta-amyloid plaques and the destruction of nerve cells.

In the last two years, biologists have made significant progress in understanding what causes this disease and what it is. For example, scientists have recently found out that Alzheimer's disease can be contagious, discovered that beta-amyloid plaques can be an important part of the innate immune system, and found several promising methods of its treatment.

One of these methods is to make the PPARGC1A protein, associated with the work of cellular self–purification systems and cellular metabolism, fight beta-amyloid plaques more actively. Sastre and her colleagues suggested that this could be done with the help of gene therapy, inserting additional copies of this gene into the cells of the hippocampus, the memory center, and into the cerebral cortex.

To test this idea, scientists raised a population of mice suffering from Alzheimer's disease, into whose brains they injected a special retrovirus containing "extra" copies of PPARGC1A. Another group of rodents, which they did not infect with the virus, made up the control group.

Four months later, the scientists compared how the condition of the mice in both groups had changed. It turned out that the appearance of additional copies of PPARGC1A had an extremely positive effect on the condition of rodents, sharply reducing the number of beta-amyloid tangles in their nerve cells and preventing the appearance of negative consequences from the development of Alzheimer's disease.

According to scientists, such mice had a good memory, and they coped with the task of finding a way out of the maze and other cognitive problems no worse than healthy rodents who did not suffer from Alzheimer's disease. The fact of their cure was confirmed by the fact that almost no cells died in their hippocampus and cortex, where mass death of neurons is usually observed during the onset of the last phases of Alzheimer's disease.

As scientists hope, their developments will serve as the basis for the development of a retroviral vaccine suitable for use among humans and does not require a potentially fatal injection of the drug directly into the brain. According to scientists, such gene therapy can be used not only for existing Alzheimer's disease, but as a kind of "vaccination" that prevents its development.

Portal "Eternal youth" http://vechnayamolodost.ru  11.10.2016