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Cistanche is very good for Alzheimer's disease

Researchers have successfully introduced a protective gene against Alzheimer's disease into human cells.

Researchers from Canada's Université Laval School of Medicine and the Université Québec-Laval Research Center have successfully edited the genomes of human cells grown in vitro, introducing a mutation that could provide patients with Alzheimer's disease. disease protection. Details of the breakthrough were recently published in The CRISPR Journal.

Research published in "The CRISPR Journal" (latest impact factor: 6.071) on February 7, 2022

"Some genetic mutations increase the risk of Alzheimer's disease, but there is one mutation that reduces this risk," said lead author Professor Jacques-P Tremblay, "a rare mutation identified in the Icelandic population in 2012. The mutation has no known disadvantages for carriers and reduces the risk of Alzheimer's disease. Using an improved version of the CRISPR gene-editing tool, we have been able to edit the genome of human cells to insert this mutation."

The brains of Alzheimer's patients have amyloid plaques that cause neurons to die due to their toxic levels. These plaques form after amyloid precursor protein is cleaved by an enzyme called beta-secretase. "The Icelandic mutation makes it harder for the enzyme to cleave the amyloid precursor protein. As a result, there is less formation of amyloid plaques," Tremblay explained.

Dr. Jacques-P Tremblay

In theory, introducing the Icelandic gene mutation into the genomes of people at risk of developing Alzheimer's disease could prevent or slow the progression of the disease. "Unfortunately, we can't go back and repair the damage that caused the neurons to die," Tremblay said, "so this treatment is especially suitable for people from families affected by this genetic disorder, a familial Alzheimer's disease. The disease manifests itself as memory problems between the ages of 35 and 40. If successful, it could also be used to treat the most common form of sporadic Alzheimer's disease, which typically develops early symptoms of the disease after age 65."

According to the researchers, the challenge now is to find a way to edit the genomes of millions of brain cells. "We are investigating different possibilities, including using non-infectious viruses to deliver editing complexes inside neurons. Now that the proof-of-concept has been established in human cells in vitro, we will be able to do this in a neuron that exhibits Alzheimer's disease. Test this method in mice. If these findings are conclusive, we hope to conduct a small study in people between the ages of 35 and 40 who have a genetic mutation that causes Alzheimer's disease, " Tremblay said.

Source：Université Laval

Another step towards gene therapy for Alzheimer's

Reference：

Tremblay G, Rousseau J, Mbakam CH, Tremblay JP. Insertion of the Icelandic Mutation (A673T) by Prime Editing: A Potential Preventive Treatment for Familial and Sporadic Alzheimer's Disease. CRISPR J. 2022 Feb 7. doi: 10.1089/crispr.2021.0085. Epub ahead of print. PMID: 35133877.