A team in California has identified the gene that is the primary risk factor for developing Alzheimer’s disease. More importantly, the scientists have reportedly created a way to correct the harmful gene in human cells.
According to researchers at Gladstone Institutes in San Francisco, people who have one copy of the “apoE4” gene are at twice the risk for developing Alzheimer’s. A person with two copies of the gene have 12 times the risk for eventually suffering from the degenerative disorder. Scientists found that apoE4 is only slightly different from the gene apoE3 however, the protein it produces within the body is much more damaging to human brain cells.
Using these findings, Dr. Yadong Huang and his team say they’ve been able to change the apoE4 genes into the harmless apoE3 gene. Huang made the breakthrough by experimenting on human stem cells instead of mice, which several Alzheimer’s researchers have done in the past. “Many drugs work beautifully in a mouse model, but so far they’ve all failed in clinical trials,” Huang said in a press release. “One concern within the field has been how poorly these mouse models really mimic human disease.”
Using pluripotent stem cells — which can develop into any kind of cell in the body — the team was able to see the effect of reducing apoE4 in brain cells for the first time. The Gladstone team has concluded that removing the apoE4 protein from the body will cut off a major risk factor for Alzheimer’s in humans.
The team’s apoE4 “structure correctors” reportedly restored normal function to the cells and wiped out any sign of the disease. The scientists are now working with the pharmaceutical industry in hopes of starting human testing in the near future.