Researchers create technology that modifies genes to potentially reduce harmful cholesterol.

Researchers have shown that in people with a genetic disease, gene editing can drastically lower excessive cholesterol levels. More patients are required for confirmation, but the experimental medication may prevent heart attacks and strokes; further study is necessary.

Professor of cardiovascular medicine at the Icahn School of Medicine at Mount Sinai in New York and director of the Mount Sinai Fuster Heart Hospital, Dr. Deepak Bhatt, says, "These data are really very exciting."

Not participating in the study, Bhatt emphasized that a great deal more research is required to address significant unanswered problems. But according to Bhatt, "this could have an enormous impact on cardiovascular disease."

Rewriting a single DNA letter in the human liver is a novel approach to treating cardiac disease, according to research published in a study by Verve Therapeutics. Although some independent experts are still apprehensive about long-term safety and other issues, the study is anticipated to have a clinical impact.

The United States loses over 695,000 persons to heart disease each year as a result of elevated low-density lipoprotein (LDL) cholesterol levels. At Verve, patients with familial hypercholesterolemia, a hereditary condition that affects 1 in 250 persons, were investigated from birth. The goal of the study was to create a one-time therapy for anyone at risk as well as a treatment for people with the hereditary disorder using gene editing.

Researchers employed CRISPR, a type of gene editing that makes it easier than ever to make precise modifications to DNA. They made use of a more recent iteration of CRISPR known as "base-editing," which allows researchers to change specific genetic code letters.

Researchers employed CRISPR, a type of gene editing that makes it easier than ever to make precise modifications to DNA. They made use of a more recent iteration of CRISPR known as "base-editing," which allows researchers to change specific genetic code letters. The continuing trial involves injecting various dosages of a CRISPR base-editing molecule to modify the PCSK9 gene, which is essential for the synthesis of LDL cholesterol.

A dose high enough to result in a favorable reduction in LDL was administered to only three participants. The prospect of novel treatments for numerous genetic disorders, such as cancer, heart disease, AIDS, Alzheimer's, and other conditions, is what has people excited about gene editing.

"There are a lot of uncertainties. This is a very bold approach but we'll have to see," Topol says.