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Researchers were able to dramatically lower levels of bad cholesterol after injecting human subjects with an experimental gene editing treatment. According to a scientific journal NatureThis is the first time this technique, called base editing, has been performed on humans.
However, at least one person died after receiving the IV, raising concerns about safety.
In the clinical trial, 10 subjects with congenitally high levels of bad cholesterol, also known as low-density lipoprotein (LDL), were given injections of VERVE-101, a gene-editing treatment that uses base-editing technology. . This treatment turns off the gene for the protein PCSK9; It is present in the liver and regulates LDL.. High levels of LDL can cause coronary heart disease.
Base editing utilizes CRISPR tools to precisely change or edit specific parts of genes. Avoid DNA duplex degradationwhich is different from previous gene editing techniques.
The results were promising, with participants seeing a 55% reduction in LDL after 28 days. The average LDL level before the experiment was 193 mg/dL, which is extremely high and potentially life-threatening. People’s LDL should be less than 100. according to medical standards.
Six months after the injection, participants who received higher doses of VERVE 101 still had lower LDL levels.
“We don’t see that with statins. We don’t see that much of a difference,” said Ritu Tanman, a cardiologist at the University of Pittsburgh in Pennsylvania. Nature. Tanman was not involved in the clinical trial.
However, this treatment also has drawbacks. In addition to temporary increases in liver enzyme levels, participants in this trial reported short-term bouts of chills, fever, and headache, similar to having the flu.
And of the 10 subjects, one subject died of a heart attack about five weeks after receiving VERVE-101, and a second subject had a non-fatal heart attack one day after the injection. Nature A safety committee of third-party experts reported that the fatal heart attacks were not caused by VERVE-101 and that they already had “advanced heart disease.”
Biotechnology company Verve Therapeutics (Boston, Massachusetts) announced that it plans to begin a Phase 2 clinical trial of VERVE-101 in 2025. Nature.
According to , VERVE 101 is composed of two molecules of RNA within a lipid nanoparticle. Nature. One molecule sniffs out her PCSK9 gene, and another RNA edits the gene itself. When VERVE 101 is injected into a human subject, the package reaches the nucleus of the liver cell, where the RNA editing molecule makes precise changes to her PCSK9 gene by changing one of its nucleotide bases. This movement promotes inactivation of the PCSK9 gene, stopping the liver from producing her PCSK9 protein and causing a reduction in LDL levels.
In other words, the work could be groundbreaking. — However, there are many unknowns about how gene editing affects other parts of the human body. The research team will spend the next 14 years tracking the surviving subjects to find out.
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