心脏病,肝脏基因 z-bo 2020-08-17 475

　　Recently, the Harvard Stem Cell Research Institute (HSCI) has collaborated with scientists at the University of Pennsylvania to reduce the function of the 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-sponsored by Krone Musnull, associate professor in the Department of Stem Cell and Regenerative Biology (SCRB) at Harvard University, and Daniel Reid of Pennsylvania. Musnur said: "In the first experiment, the results were very good." From the laboratory to the human clinical trials, it takes about 10 years to treat heart disease with this method. Have.

　　In 2003, a French research team found that a family with high cholesterol suffers from heart disease very quickly, and that PCSK9 mutations can cause high cholesterol and heart attacks. Another American team found that about 3% of PCSK9 gene mutation patients have the opposite effect: their low-density lipoprotein (LDL) cholesterol levels are 15% to 28% higher than average. It is low and the risk of heart attack is 47%-88% lower than the average.

　　"We speculate that nature did experiments and someone won a genetic mutation award. Since then, we have no protection, no heart attack, no side effects." Musnur said, "If we can reproduce this, sudden mutations are okay. Protect many people and reduce the risk of heart attack. Variation genes: "The PCSK9 gene is mainly expressed in the liver. "The protein produced is active in the blood. It prevents the blood from removing cholesterol. Several pharmaceutical companies have developed antibodies against this protein. Antibody drugs will not last forever. An injection is required every few weeks. The next step is to take it. Statins (such as Lipitor), but many people who take this drug every day have a heart attack and require other methods."

　　"Permanent changes to the genome to use gene editing technology. Through genome editing, normal people look like people born with good mutant genes. In experiments, the delivery system enters the mouse liver 3-4 days after all. Hepatocytes are damaged by most copies of the PCSK9 gene and lose their activity. "Another result is that the cholesterol levels in the 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 in their 40s, half of men and a third of women suffer from heart disease." Musnur said it is safe, at least theoretically it can be used as a vaccine. When used in a crowd, it can reduce the risk of heart attack by 30%, 50% or 90%. "