(NewsNation) — Cardiovascular disease is the leading cause of death in the U.S., and for the first time, researchers believe they’ve produced a way to rewrite DNA in liver cells to reduce cholesterol levels.
During this year’s American Heart Association annual conference, doctors revealed a technique for rewriting the genetic code directly in the body that slashed “bad” cholesterol levels in three people, according to Science. The three people were prone to dangerously high levels of the artery-clogging fat.
The clinical trial, the biotech Verve Therapeutics, used a technique that relied on a blood infusion of a so-called base editor, designed to disable a liver protein, PCSK9, that regulates cholesterol, Science reports.
Researchers believe it’s an approach that is more precise, and possibly safer, than disrupting a gene with CRISPR, the gene-editing tool from which base editing is derived.
These results mark the first time this CRISPR variant has been infused into people to treat a disease, according to Science. It’s also a proof of principle for using gene editing for a common health problem like high cholesterol rather than a rare disease.
Dr. Dave Montgomery, a board-certified cardiologist and NewsNation medical contributor, explained that researchers are using base editing, by taking out one abnormal letter or base pair and then bringing back in another one.
“There’s millions of letters on a gene, there’s one letter that’s there. So imagine how difficult that is to find, this machinery finds that place, cuts it, and then helps the body use its own natural repair mechanisms. Then the product is a normally functioning gene,” he said.
He broke down this example: “In the cholesterol abnormalities, in sickle cell, in cardiomyopathy, in various cancers and HIV there are these mutations that we could, if the mutations are repaired, we could cure the disease.”
Montgomery said experiments have been started, and researchers are seeing a lot of good data, but it could be years before a DNA-altering drug could hit the market.
Verve Therapeutics hopes its base editor could eventually be a one-time solution for tens of millions of middle-aged people who are struggling to control their cholesterol with daily drugs.
