Gene therapy of Alzheimer's disease and other neurodegenerative diseases?
Gene therapy protects mice from dementia
Copper newsGene therapy helped mice get rid of a pathological protein in nerve cells, the accumulation of which leads to the development of Alzheimer's disease, reports Medical News Today (Gene Therapy Boosted Brain Cell Disposal Of Toxic Proteins And Protected Mice From Alzheimer's).
A report on a study by a group of American scientists led by Charbel Moussa from the Georgetown University School of Medicine is published in the journal Human Molecular Genetics (Parkin mediates beclin-dependent autophagic clearance of defective mitochondria and ubiquitinated A[beta] in AD models).
For the study, genetically modified mice were grown, which by the age of six months had accumulations of the pathological beta-amyloid protein in neurons and amyloid plaques on the surface of these cells. Such animals are used as laboratory models of Alzheimer's disease.
Moussa and his colleagues studied the action of the parkin protein, which is one of the components of the enzymatic complex that ensures the breakdown of proteins in cells. With the help of a harmless form of the immunodeficiency virus, scientists have introduced several copies of the gene responsible for the synthesis of parkin into the genome of mouse neurons.
According to the study, an increase in parkin production by one and a half times led to a decrease in the amount of beta-amyloid in neurons and on their surface. In addition, due to the excess of parkin, the mechanism of decay of damaged mitochondria was triggered, as a result of which the processes of intracellular oxidation were normalized, and the synthesis of neurotransmitters providing the transmission of a nerve impulse was restored.
Comparing mice that underwent gene therapy with animals from the control group, the scientists found that the amount of beta-amyloid decreased by 75 percent. Similarly, the proportion of dead neurons decreased in rodents.
Thus, scientists were able to demonstrate that an increase in the synthesis of parkin in neurons not only causes the breakdown of beta-amyloid in nerve cells, but also leads to the restoration of the function of neurons disrupted as a result of the accumulation of pathological protein.
The authors intend to reproduce the results obtained during experiments on patients with neurodegenerative diseases, including Alzheimer's, Parkinson's and Huntington's diseases.
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