Zinc fingers against dementia
Researchers at Massachusetts General Hospital in Boston used gene therapy to reduce the level of tau protein - a protein that accumulates in the brains of patients suffering from Alzheimer's disease – on an animal model of this condition.
The new strategy includes a technology of regulation using transcription factors – zinc finger proteins (ZFP-TFs), which are DNA-binding proteins and can be used to control the expression of certain genes.
A complex of three "zinc fingers" (blue) and DNA (red). Zinc ions are shown in green.
In this study, the therapy was aimed at suppressing the expression of the gene encoding tau protein. Mice with Alzheimer's disease received a single injection of an adeno-associated vector carrying ZFP-TFs directly into the hippocampus area or intravenously. This led to a persistent (up to 11 months) decrease in the level of tau protein in the brain by 50-80%.
The overall behavior of the animals in the study was evaluated daily. There were no obvious changes in body weight, behavior (for example, nutrition, care, social behavior, spontaneous movements, mobility, sleep) or the physical state of health of the mice.
It is important to note that the therapy helped to eliminate damage to neurons around amyloid plaques that were present in animal brain cells. Tau protein reduction during therapy was not accompanied by side effects.
Simplicity – a single introduction of gene-regulatory therapy instruments by intravenous injection – makes this strategy especially attractive. There is a lot of work to be done to study the safety of therapy before using it to treat people, but a promising first step has already been taken.
Article by S.Wegmann et al. Persistent repression of tau in the brain using engineered zinc finger protein transcription factors is published in the journal Science Advances.
Aminat Adzhieva, portal "Eternal Youth" http://vechnayamolodost.ru based on the materials of Massachusetts General Hospital: Gene therapy using "zinc fingers" may help treat Alzheimer's disease, animal study shows.