Alzheimer's gene neutralised in human brain cells for the first time
• Henry Bodkin
9 April 2018 • 8:05pm

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.

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.

Having one copy of the apoE4 gene more than doubles a person’s likelihood of 
developing Alzheimer’s disease, whereas having two copies increases the risk 

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.

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.

Last month senior British scientists predicted that within the next few decades 
Alzheimer’s sufferers will be able to live with the disease without the 
devastating symptoms.
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