A Single Infusion of a Gene-Editing Treatment Lowered High Cholesterol

In a small initial test in humans, researchers showed that a single infusion of a new gene-editing treatment can reduce cholesterol, the fatty substance that clogs and hardens arteries over time.

The experiment was conducted on 10 participants with a hereditary disease leading to extremely high levels of LDL or “bad” cholesterol, which can lead to heart attacks at an early age. Even though they were taking medication to lower cholesterol, the volunteers already had heart disease. They participated in a trial conducted in New Zealand and the United Kingdom by Verve Therapeutics, a biotechnology company based in Cambridge, Massachusetts.

Gene editing treatment aims to permanently lower cholesterol levels using Crisp to modify a gene in the liver. The researchers administered a single infusion, at varying doses, to patients whose average age was 54 years. Although the lowest doses didn’t have much effect, the highest dose reduced LDL cholesterol by 55 percent in the single patient who received it. Meanwhile, two patients who received the highest dose saw reductions of 39 percent and 48 percent.

The first patient was treated just six months ago and researchers are still following all participants. Preliminary results were presented at the American Heart Association annual meeting in Philadelphia on November 12.

Gene editing could provide a more sustainable option for treating hereditary high cholesterol, which today requires long-term medication. “Current care consists of daily pills and intermittent injections that must be taken for decades. This places a very heavy treatment burden on patients, providers and the healthcare system,” Andrew Bellinger, chief scientific officer of Verve Therapeutics, said at a press conference this weekend.

But no one has ever used gene editing to lower cholesterol levels in humans before. “This is a strategy that could be revolutionary,” Karol Watson, a cardiologist at UCLA, said at the news conference. “But we need to make sure it’s safe.”

The treatment uses a newer, more accurate form of Crispr called basic edition to inactivate a gene in the liver called PCSK9. This gene plays an essential role in controlling LDL cholesterol in the blood. Instead of cutting genes, as Crispr is designed to do, base editing simply swaps one letter of DNA for another. Verve’s processing is designed to change an A to a G, effectively transforming the PCSK9 disabled gene.

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