Researchers have announced an important breakthrough in the battle against Alzheimer's after neutralizing the most significant gene responsible for the disease.
A team in California successfully identified a protein associated with the apoE4 gene, which is associated with a high risk of disease, and then managed to prevent its damaging effects on human neuronal cells.
The study may open the door to a new drug that can stop the disease, but scientists have urged caution, since until now their compound has only been tested in cell cultures in the laboratory.
Having one copy of the apoE4 gene more than doubles the likelihood that a person will develop Alzheimer's disease, while having two copies increases the risk 12-fold. Previous research has shown that about one in four people carry this gene.
In human neurons, the protein produced by the apoE4 gene cannot function properly and is broken down in cells into disease-causing fragments. This leads to several of the problems that are commonly found in Alzheimer's disease, which affects 7.1 percent of Britons over 65, including the accumulation of protein tau and amyloid peptides.
A team of researchers at the Gladstone Institute set out to determine whether the presence of the protein was causing the cell damage.
Using stem cell technology, the scientists created skin cell neurons from Alzheimer's patients with two copies of the apoE4 gene.
By comparing the grown cells with those that did not produce the APOE protein, they concluded that the mere presence of the apoE4 protein caused brain damage. They then applied a genetic structure corrector that eliminated the signs of Alzheimer's.
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Researchers are now working with the pharmaceutical industry to improve the compounds used so they can be tested in living people.
The significance of the experiment is especially great because it was carried out on human cells.
Many previously developed drugs worked well in mice, but so far all of them have failed in clinical trials. One problem in this area is how well these mouse models actually mimic human disease.
Therefore, the researchers started experimenting directly on human brain cells because they realized that the presence of the apoE4 gene does not alter the production of amyloid protein in the mouse brain.
Leading British scientists predicted last month that Alzheimer's sufferers would be able to live with the disease over the next several decades without devastating symptoms.
The study was published in the journal Nature Medicine.
Anton Komarov