Breakthrough Gene treatment in Alzheimer’s announced by Scientists


As per an article published in Telegraph, Scientists have claimed an important breakthrough in the battle against Alzheimer’s after neutralising the most significant gene responsible for the disease for the first time.

The article highlights that a team in California successfully identified the protein associated with the high-risk apoE4 gene and then managed to prevent it damaging human neuron cells. The study could open the door to a potential new drug capable of halting the disease, however the researchers have urged caution because so far their compound has only been tried on collections of cells in a laboratory.

Previous studies have indicated that roughly one in four people carry the gene. In human neurons, misshapen apoE4 protein cannot function properly and is broken down into disease-causing fragments in the cells. This results in several of the problems commonly found in Alzheimer’s disease, which affects 7.1 per cent of Britons above the age of 65, including the accumulation of protein tau and amyloid peptides. The team at Gladstones Institutes set out to establish whether the presence of the protein was causing the damage, or whether a lack of it was to blame.

The article underlines that using stem cell technology, they created neurons from skin cells donated by Alzheimer’s patients with two copies of the apoE4 gene. By comparing the cells with those which did not produce an apoE protein they concluded that it was the the mere presence of the apoE4 protein was causing brain damage. They then applied a genetic “structure corrector”, which eliminated the signs of Alzheimer’s. The researchers are now working with the pharmaceutical industry to improve the compounds so they can be tested on human patients. The experiment is particularly significant because it took place in human cells.

Yadong Huang, who led the study, which is published in Nature Medicine, said: “Drug development for Alzheimer’s disease has been largely a disappointment over the last 10 years. Many drugs work, beautifully in a mouse model, but so far they’ve all failed in clinical trials. One concern within the field has been how poorly these mouse models really mimic human disease.”

Huang and his colleagues went straight for human brain cells rather than the traditional mouse trial because they realised the presence of the apoE4 gene does not change the production of amyloid beta in a mouse brain.


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