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| Photo by Sangharsh Lohakare on Unsplash |
Sometimes, the most groundbreaking ideas in medicine begin with an observation in nature. In this case, researchers noticed a small group of people who, because of a genetic quirk, naturally have very low cholesterol and almost no risk of heart disease. Their bodies simply don’t activate a gene called ANGPTL3, which influences how the liver handles cholesterol and triglycerides. And these individuals live with that protection—without taking any medication.
That discovery became the spark for a new wave of gene-editing research. Now, in a very early clinical study released in The New England Journal of Medicine and presented at the American Heart Association’s annual meeting, scientists have shown that a single CRISPR-based infusion can reduce LDL (“bad”) cholesterol by about 50% and triglycerides by an average of 55% at the highest tested dose. That’s a remarkable shift—especially considering that the treatment may be permanent.
The study, led by preventive cardiologists at the Cleveland Clinic, involved just 15 participants who had dangerously high cholesterol despite standard therapy. The research team, including senior investigator Dr. Steven Nissen, described the finding as something that once felt impossible:
“If you’d asked me 15 years ago if we could have done something like this, I would have thought you were crazy.”
How the Treatment Works: Editing the Liver, Not the Whole Body
The therapy uses CRISPR-Cas9, the gene-editing tool often described as “molecular scissors.” The idea is elegantly targeted: send the treatment to the liver—the organ responsible for producing and clearing cholesterol—and disable the ANGPTL3 gene there. Unlike people who are born with the mutation in every cell, this approach focuses only on the liver, which experts say may help limit unintended effects elsewhere in the body.
Importantly, researchers saw no serious treatment-related safety concerns in the trial. Side effects were mostly mild, such as local infusion irritation. One participant died months later, but investigators emphasized that this person received the lowest dose—far too low to have had any effect—and had severe underlying cardiovascular disease. To ensure long-term safety, participants will be followed for 15 years, which is standard when genetic material is altered.
If We Already Have Powerful Cholesterol Drugs, Why Does This Matter?
This is where nuance is essential. As Dr. Pradeep Natarajan, director of preventive cardiology at Massachusetts General Hospital, noted in CNN’s coverage, today’s medications—including statins and injectable PCSK9 inhibitors—can already lower cholesterol to levels similar to those seen in the study, and in some cases, even lower.
So the breakthrough here is not about achieving new low cholesterol records.
The real shift is eliminating the need for lifelong treatment.
As preventive cardiologist Dr. Ann Marie Navar explained, many people simply struggle to stay on their medications—even when the stakes are life-threatening. Some forget, some dislike side effects, and others lose access or insurance coverage.
This is why experts describe the “one-and-done” model as potentially transformative—it turns cholesterol management from something you maintain daily into something you correct once.
Still, cardiologist Dr. Eric Topol cautioned in NPR’s reporting that gene editing remains expensive and long-term effects are still unknown. For now, it remains a promising concept, not a replacement for current therapy.
The Big Question: Could This Actually Prevent Heart Attacks?
Not yet — that’s still unknown. This early study measured cholesterol levels, not heart attack or stroke rates. Larger and longer trials are already being planned to answer that question directly.
The most likely early candidates for the treatment are younger patients with genetic cholesterol disorders, who face dangerously high risk throughout their lives.
If future trials confirm what we’re seeing now, the implications could reach millions.
What I’ve Learned Along the Way
This moment reminds me of the early 2000s, when statins were still being debated. Some experts were enthusiastic, others cautious, and no one could yet see how profoundly they would reshape cardiovascular medicine. We’re in a similar place now—right at the edge of possibility, before clarity settles in.
Wellness culture often tells us high cholesterol is about willpower. Eat cleaner. Run more. Be better. But genetics have a larger voice than most people realize. Some bodies simply make more cholesterol, no matter how perfect the diet.
Here’s the clearest insight research keeps reinforcing: The biggest health gains come from lowering LDL early and keeping it low consistently. Whether that’s through lifestyle, medication, or one day gene editing, the goal is the same.
If this new approach holds up, it won’t just change cholesterol treatment. It may change our relationship to chronic disease itself—moving us from lifelong management to long-term prevention.
And that’s a future worth watching.