Of course, it will take time to see how effective the modification of liver DNA will ultimately be in fighting cholesterol.
A patient from New Zealand volunteered to become the first person in the world to receive gene-editing therapy, with the aim of lowering cholesterol levels in his blood. The first experiments on monkeys had already given promising results.
Going into detail, the patient participates in a clinical trial of US biotech company Verve Therapeutics and suffers from heart disease, having inherited the risk for excessively high cholesterol, which in turn increases the risk of heart attacks and heart disease.
His treatment involved injecting into his body a version of the CRISPR gene-editing tool designed to slightly modify the DNA in liver cells. In this case the gene modification targets a single letter in the liver’s DNA code, specifically the PCSK9 gene. What is sought is the reduction of the levels of LDL cholesterol, the so-called “bad” cholesterol.
Speaking about it, Mr gene researcher who founded Verve three years ago and is now its CEO, Sekar Kathiresan, said: “If this works and it’s safe, that’s the answer to heart attack — that’s the cure.”
In case you don’t know, LDL cholesterol is a fatty molecule that can clog arteries and cause them to harden, leading to coronary heart disease, one of the leading causes of death worldwide. The most widely prescribed drugs can cut cholesterol levels in half, but they come with side effects that may be too much for some patients.
Focusing on the potential benefits of gene editing, Verve consultant Eugene Braunwald said: “Gene editing is the magic wand because it’s done once and for all. You never have to do anything again. It’s incredibly important because cardiovascular disease is the most common cause of death in the industrialized world, and LDL is the leading cause.”
Of course, it will take time to see how effective liver DNA modification will ultimately be in fighting cholesterol, but using the technology can certainly bring about dramatic changes.
Verve now hopes to work with another 40 adult patients with heterozygous familial hypercholesterolemia (HefH), an inherited genetic disorder that causes dangerously high cholesterol levels, to better assess the safety and effectiveness of its injection.
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