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Gene-editing treatment shows promise in reducing cholesterol levels

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Gene-Editing,Treatment,Cholesterol

Researchers have conducted a small initial test in people, demonstrating that a single infusion of a gene-editing treatment can reduce cholesterol levels. The experiment involved 10 participants with high LDL cholesterol levels, who were already suffering from heart disease. The treatment, which uses Crispr to edit a gene in the liver, aims to permanently lower cholesterol. While lower doses had minimal effects, the highest dose reduced LDL cholesterol by 55 percent in one patient. Two patients who received the next-highest dose saw reductions of 39 percent and 48 percent.

In a small initial test in people, researchers have shown that a single infusion of a novel gene-editing treatment can reduce cholesterol, the fatty substance that clogs and hardens arteries over time. The experiment was carried out in 10 participants with an inherited condition that causes extremely high LDL, or “bad,” cholesterol levels, which can lead to heart attack at an early age. Despite being on cholesterol-lowering medications, the volunteers were already suffering from heart disease.

They joined a trial in New Zealand and the United Kingdom run by Verve Therapeutics, a Cambridge, Massachusetts–based biotech company. The gene-editing treatment aims to permanently lower cholesterol by using Crispr to edit a gene in the liver. Researchers gave a single infusion, in varying doses, to the patients whose average age was 54. While the lower doses didn’t have much of an effect, the highest dose reduced LDL cholesterol by 55 percent in the single patient who received it. Meanwhile, two patients who got the next-highest dose saw reductions of 39 percent and 48 percent

 

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