For the first time, gene editing provides guidance on lowering cholesterol levels.

Researchers have presented evidence that gene editing can reduce cholesterol levels, a major risk factor for the leading cause of death in the country.

Preliminary results from a study involving 10 patients born with a genetic disorder causing very high cholesterol levels show that gene editing inside the liver can significantly reduce levels of "bad cholesterol."

The experimental treatment needs to be tested on a larger number of patients, with longer follow-up, to confirm its safety and effectiveness. However, the results are presented as potential groundbreaking evidence for a concept that could ultimately provide a powerful new way to prevent heart attacks and strokes.

"These data are really very interesting," says Dr. Dipak Bhatt, director of cardiology at Mount Sinai Hospital and professor of cardiovascular medicine at the Icahn School of Medicine at Mount Sinai in New York.

Bhatt, who did not participate in the research, emphasized that much more research is needed to address important open questions. However, "this could have a huge impact on cardiovascular diseases," says Bhatt.

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New method for treating cardiovascular diseases

The long-awaited data from a study conducted by Verve Therapeutics, Inc. in Boston were presented on Sunday at the American Cardiology Association meeting in Philadelphia.

"What we're trying to do is develop an entirely new way to treat cardiovascular diseases," said Dr. Sekar Kathiresan, CEO of Verve, in an interview with NPR. "We are very excited. This is the first evidence in history that you can actually rewrite one letter of DNA in the human liver and get a clinical effect. So we're excited."

But some other independent scientists also remain cautious.

"I hope it works. But there are many aspects that are still unclear," including long-term safety, says Dr. Eric Topol, a cardiologist and professor of molecular medicine at Scripps Research in California. "There is significant uncertainty here."

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Heart diseases kill about 695,000 people every year in the United States.

High cholesterol levels, known as low-density lipoprotein cholesterol (LDL-C), narrow arteries, blocking blood flow to the heart and brain and creating conditions for heart attacks and strokes.

Verve studied patients born with familial hypercholesterolemia, a genetic disorder that affects about 1 in 250 people and causes extremely high levels of LDL-C.

Patients can take medications, including the popular class of statins, to reduce LDL-C levels. But while these drugs are very safe and effective, many patients do not take them regularly as needed, making them vulnerable. Therefore, there is hope that gene editing treatment can not only cure people with this genetic condition but also provide a one-time therapy for everyone at risk.

"The same drug should be useful for any regular patient without a genetic condition," says Kathiresan.

Researchers used a form of gene editing known as CRISPR, which allows scientists to make very precise changes to DNA much easier than ever before. In particular, they used a new version of CRISPR known as "base editing," which allows scientists to rewrite individual letters of the genetic code.

In this case, editing occurred in the liver cells inside the organ. Other gene editing approaches required removing cells from the body, editing them in the laboratory, and then reintroducing them to patients.

As part of the ongoing trial, scientists are introducing various doses of the CRISPR base-editing molecule into the liver to edit the PCSK9 gene, which is necessary for the production of LDL-C.

Of the 10 patients treated so far, aged 29 to 69, all were in the UK and New Zealand. Only three patients received a high enough dose to cause a favorable reduction in LDL-C levels. Two patients showed a reduction of 39% and 48% in LDL-C levels. One patient who received the highest dose showed a 55% reduction in LDL-C levels, which has persisted for at least six months.

"This is really the first evidence that you can actually rewrite one letter of DNA in the liver of a living person and get a clinical effect," says Kathiresan.

Mohammed Khan, 47, from London, was one of the latest patients to undergo treatment in the study. Khan's father died at the age of 42, and his two older brothers had already suffered heart attacks. Khan had already undergone a procedure for narrowing heart arteries.

"This is a brilliant approach," Khan said in an interview with NPR. "It's fantastic. I hope for the best. I am very, very optimistic that it will have an effect on my body."

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Cholesterol treatment is just the beginning

Gene editing has caused a huge sensation as this method could lead to the development of new treatments for many genetic diseases, including cancer, heart disease, AIDS, Alzheimer's disease, and other conditions.

In fact, the Food and Drug Administration is ready to approve the first gene editing method for the treatment of the devastating blood disease sickle cell anemia next month.

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