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Researchers have been able to significantly lower bad cholesterol levels in humans after undergoing an experimental gene-editing treatment. As a science journal NatureThis is the first time, so-called base editing, has been done on humans.
But at least one person died after receiving blood, prompting safety concerns.
In the clinical trial, 10 people with naturally high levels of bad cholesterol, aka low density lipoprotein (LDL), were given VERVE-101, a gene-editing therapy that uses a basic editing technique. This treatment inactivated the gene for the protein PCSK9. It is found in the liver and regulates LDL. High levels of LDL can cause cardiovascular disease.
Base editing uses CRISPR tools to change or edit specific parts of a gene in precise movements. To prevent DNA double strands from breaking apartUnlike previous gene editing methods.
The results were promising, with test subjects’ LDL dropping by 55 percent after 28 days. Before the test, they had an average LDL of 193 mg/dL, which is sky-high and life-threatening. People should have an LDL below 100; According to medical standards.
Six months after the injection, participants who received the highest dose of VERVE 101 still had lower LDL levels.
“We don’t see that with statins — we never see that much of a difference,” said Ritu Thaman, a cardiologist at the University of Pittsburgh in Pennsylvania. Nature. Teman was not involved in the clinical trial.
But the treatment has its drawbacks. Participants in the trial reported short-term chills, fever and headache — similar to those experienced by the flu — in addition to a temporary increase in liver enzyme levels.
And of the 10 test patients, one subject died of a heart attack five weeks after receiving VERVE-101, while the second subject had a non-fatal heart attack one day after the injection. Nature Third-party experts said the safety panel said the fatal heart attack was not caused by VERVE-101 and that he was already suffering from “severe heart disease.”
Verve Therapeutics, a Boston, Massachusetts-based biotech firm, plans to begin a Phase 2 clinical trial of VERVE-101 in 2025. Nature.
VERVE 101 is composed of two RNA molecules in a lipid nanoparticle; Nature. One molecule knocks out the PCSK9 gene, while the other RNA modifies the gene itself. When VERVE 101 is injected into a human test subject, the package travels to the nuclei of the liver cells, where the RNA editing molecule changes one of its nucleotide bases, causing a precise change in the PCSK9 gene. This action triggers the inactivation of the PCSK9 gene, causing the liver to stop making PCSK9 proteins, leading to lower LDL levels.
The work can be the basis, in other words – But there are also many unknowns about how gene editing affects the rest of the human body. The research team followed the surviving test subjects for the next 14 years.
More on gene editing: A monkey can live for two years with a genetically engineered pig kidney