动物实验,肝脏基因 z-bo 2020-08-31 408

　　Recently, the Harvard Stem Cell Research Institute (HSCI) has collaborated with scientists at the University of Pennsylvania to alter the function of a liver gene called PCSK9 through genome editing technology to reduce the risk of heart attack. A network report organized by physicists shows that a single injection can permanently reduce cholesterol levels in mice and reduce the risk of heart attack by 40-90%.

　　This work was co-authored by Krone Musnull, associate professor of Stem Cell and Regenerative Biology (SCRB) at Harvard University and Danieleid of Pennsylvania. Musnur said: "In the first experiment, this result was very good." From the laboratory to the human clinical trial, the process of using this method to treat heart disease takes about 10 years. Have.

　　In 2003, a French research team found that a family with high cholesterol suffered from heart disease very quickly, and found that the PCSK9 mutation caused high cholesterol and a heart attack. Another American team found that about 3% of PCSK9 mutation patients had the opposite effect: their low-density lipoprotein (LDL) cholesterol levels were 15% to 28% higher than average. Low, the risk of heart attack is 47%-88% lower than the average.

　　"I guess this experiment was done naturally, and someone won the gene mutation award. Since then, there are no protective measures, no heart attacks, no side effects." It can also protect many people and reduce the risk of heart attacks. "

　　Sulu and Reed (Reed) used a genome editing technology called CRISPR/Cas9 to "normally defect" the normal PCSK9 gene. Changed to variant genes. The produced protein "PCSK9 gene is mainly expressed in the liver" is active in the blood and can prevent the elimination of cholesterol in the blood. Several pharmaceutical companies have developed antibodies against this protein. Antibody drugs will not last forever. It needs to be injected every few weeks. The next step is to take a statin drug (such as Lipitor), but many people who take this drug every day have a heart attack and require other methods. "

　　"To permanently change the genome using gene editing technology. Genome editing makes it look like a normal person has been born with a good mutant gene. In the experiment, 3-4 days after the delivery system enters the mouse liver, all liver cells are affected. Most copies of the PCSK9 gene are destroyed and lose their activity. "Another result is that the cholesterol levels in mice are reduced by 35% to 40%. "This will reduce the human risk of heart disease by 90%.

　　"Heart disease is the killer of all mankind. Among people over 40, half of men and one-third of women suffer from heart disease." Moreover, it is absolutely safe, at least safe. In theory, it can be used as a vaccine. When used in a crowd, it is expected to reduce the risk of heart attack by 30%, 50% or 90%. "