2023-07-17 06:27:00
Genetic scissors/News1
American scientists are working on a cure for Alzheimer’s disease using CRISPR, a third-generation gene editing technology. It is believed that the accumulation of amyloid protein can be prevented by cutting or editing genes that increase the risk of developing neurodegenerative diseases such as dementia.
On the 16th (local time), according to Pier Pharma and STAT, an American biopharmaceutical media outlet, at the ‘2023 Alzheimer’s Association International Conference (AAIC)’ held in Amsterdam, the Netherlands, a research abstract on Alzheimer’s treatment using CRISPR genetic scissors was presented. Two were released. CRISPR gene scissors consist of guide RNA that searches for specific DNA and binds like a zipper, and Cas9, an enzyme protein that cuts off the binding site. The part cut out by genetic scissors was replaced with a normal gene, drawing attention as a way to treat the root of genetic disease.
When amyloid beta (Aβ) protein in the brain accumulates and clumps together in brain neurons, sticky plaques are formed, and these plaques are known to cause inflammation and cause Alzheimer’s disease. Rechembi (ingredient: recanemab), a dementia treatment recently approved by the U.S. Food and Drug Administration (FDA), was also developed on the principle of slowing down the progression of dementia by removing this amyloid beta protein.
A research team at the University of California, USA paid attention to APP, an amyloid precursor protein gene that produces this amyloid beta protein. When APP overproduces amyloid beta protein, plaques are formed, but when APP is cut with gene editing technology, amyloid beta protein is also reduced.
“There is no dispute that the APP gene plays a central role in Alzheimer’s disease,” said researcher Brandt Allston.
In a mouse experiment, the researchers said that when they cut off the end of the APP gene in mice suffering from Alzheimer’s disease using CRISPR technology, they confirmed that the amount of amyloid protein was significantly reduced. “We have confirmed that CRISPR technology is a safe and effective treatment for mice,” said Allston. “We need to test APP CRISPR technology in human clinical trials in the future.” They plan to continue their research to figure out the exact location to cut the gene.
Another study focused on ‘APOE-E4’, a mutated gene known to be a potent inducer of dementia. APOE is a transporter of lipids and cholesterol in the body, and has three genotypes: E2, E3, and E4. Among them, it is known that the risk of developing dementia increases 3 to 12 times in people with E4. In particular, people with two E4 genes are 8 to 12 times more likely to develop dementia.
A research team at Duke University in the US is conducting research on reducing this APOE-E4 using CRISPR technology. As a result of preclinical experiments with brain organoids made from human induced pluripotent stem cells (hiPSC) of Alzheimer’s patients and a humanized mouse model, APOE-E4 levels were significantly reduced.
“Innovative new ideas like CRISPR are welcome as a treatment for Alzheimer’s disease,” said Dr. Maria Carrillo, Chief Scientific Officer of the Alzheimer’s Association. “For ultimate treatment and prevention, studies applying CRISPR technology should be further expanded.”
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