Description: Scientists have claimed an important breakthrough in the battle against Alzheimer&rsquo;s after neutralising the most significant gene responsible for the disease for the first time.
Keywords: Science,Stem Cells,Health,Pharmaceutical industry,Alzheimer's Disease,Standard

# Alzheimer's gene neutralised in human brain cells for the first time

The researchers targeted the apoE4 protein Credit: Dr Robert Friedland

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 manage 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 12-fold.

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.

FAQ | Dementia

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.

### Related Topics

- Stem Cells
- Pharmaceutical industry
- Health
- Alzheimer's Disease
- Show more

### Follow the Telegraph

- Follow on Facebook
- Follow on Twitter
- Follow on Instagram

If you would like to add a comment, please register or log in
Register Log in
Please review our commenting policy

## Science latest

1. 09 Apr 2018, 8:04pm

   ### Ancient fragment of finger bone suggests humans travelled east from Africa earlier than thought

2. 09 Apr 2018, 5:00pm

   ### Cancer cure hope for endangered Tasmanian devil

3. 09 Apr 2018, 4:00pm

   ### Eyebrows crucial to human evolution - it's how we first learnt to communicate

4. 08 Apr 2018, 6:23pm

   ### Tea or a pill preferred over running to combat high blood pressure risk

5. 08 Apr 2018, 4:15pm

   ### IVF children at risk of heart disease if procedure too intense, experts warn

6. 07 Apr 2018, 12:57pm

   ### Do you have a distorted view of the world? Why life is better than we think

   Premium
7. 07 Apr 2018, 9:32am

   ### Baby wet wipes 'cause food allergy', new study warns

8. 00:42
   05 Apr 2018, 7:02pm

   ### Virgin Galactic completes first powered flight since fatal crash in 2014

9. 05 Apr 2018, 6:47pm

   ### Neanderthals were capable of ‘turbo breathing’, according to new study of their facial features

10. 05 Apr 2018, 5:00pm

    ### Brain never stops growing, scientists find, as elderly shown to have same number of new neurons as teens

11. 04 Apr 2018, 6:41pm

    ### Young heavy metal fans five times more likely to self-harm or attempt suicide

12. 04 Apr 2018, 6:34pm

    ### 10,000 black holes exist in center of Milky Way, scientists find

13. 04 Apr 2018, 6:25pm

    ### Can you spot the baffling letter in this headline that 72 per cent of people miss?

14. 04 Apr 2018, 6:16pm

    ### Women still crippled by self-doubt even when they are as smart as men, study suggests

15. 04 Apr 2018, 11:45am

    ### Is Hawkins Laboratory real? The bizarre conspiracy theory that inspired Stranger Things

    Premium
16. 04 Apr 2018, 12:01am

    ### Sugary drinks ban looming for NHS hospitals as one third fail to sign up to reduction scheme

17. 03 Apr 2018, 6:15pm

    ### Galaxy-hunting tech used to stop poachers hunting endangered animals

18. 03 Apr 2018, 5:04pm

    ### Losing life savings in middle age increases risk of early death by 50 per cent

19. 03 Apr 2018, 2:43pm

    ### Scrap the spiraliser. Pasta can help you lose weight, major review finds

    Premium
20. 03 Apr 2018, 4:09am

    ### Antarctic ice sheet loses area the size of London from base, say scientists

21. 02 Apr 2018, 11:09am

    ### Tiangong-1: China's space station breaks up over over Pacific Ocean

### We've noticed you're adblocking.

We rely on advertising to help fund our award-winning journalism.

We urge you to turn off your ad blocker for The Telegraph website so that you can continue to access our quality content in the future.

Thank you for your support.

Need help?

Click here for instructions