New DNA-Based Therapy Can Help Lower 'Bad Cholesterol' Without Statins, Finds Study

Scientists may have found a powerful new way to lower "bad" cholesterol, which did not involve the use of statin medicines. In a recent study, researchers used tiny DNA-based molecules to cut levels of LDL (bad) cholesterol by nearly 50% in animal models. This was done without the side effects that are often linked to statins. If these results stay consistent in large human trials, the therapy could become an important option for people who cannot tolerate statins or who still have high cholesterol despite taking them. The study was led by Carles J. Ciudad and Veronica Noe from the University of Barcelona's Faculty of Pharmacy and Food Sciences and the Institute of Nanoscience and Nanotechnology (IN2UB), working with Nathalie Pamir at the University of Oregon in Portland (United States). It was published in the journal Biochemical Pharmacology.

High LDL cholesterol is one of the biggest risk factors for heart attacks and strokes because it leads to the build up fatty plaques in arteries. Drugs like statins work well for many, but some people suffer from muscle aches, digestive issues, or liver problems and have to stop them. However, the new approach is different. Instead of changing how the liver handles fats, it targets a specific protein in the blood that controls how much LDL stays circulating.

What Does The New Therapy Do?

The new therapy focuses on a liver-made protein called PCSK9. This protein normally reduces the number of LDL receptors on liver cells, which means the liver can remove less bad cholesterol from the bloodstream. When PCSK9 is too active, LDL cholesterol tends to stay high, increasing the risk of heart disease.

Researchers designed small DNA-based molecules called polypurine hairpins (PPRH) that bind to the gene that produces PCSK9 and "silence" it. By reducing PCSK9 production, more LDL receptors stay active on liver cells, allowing the liver to remove more bad cholesterol out of the blood and clear it from the body. The therapy helps the body regulate cholesterol more naturally, instead of just blocking cholesterol production with a pill.

Study Findings

In experiments on human liver cells grown in the lab, one of the DNA molecules, called HpE12, reduced PCSK9 RNA by about 74% and the PCSK9 protein by around 87%. This drop suggests the molecule can effectively shut down much of the protein's production at the genetic level.

In mice carrying the human PCSK9 gene, a single injection of HpE12 reduced blood PCSK9 levels by about 50% and cut total cholesterol by nearly 47% within just three days. These early results are impressive, especially because they involve a relatively low-dose, one-time or short-course treatment rather than a daily pill.

Professor Veronica Noe said, "The results show that both HpE9 and HpE12 are highly effective in HepG2 cells. HpE12 decreases PCSK9 RNA levels by 74% and protein levels by 87%. In the case of transgenic mice, a single injection of HpE12 reduces plasma PCSK9 levels by 50% and cholesterol levels by 47% on the third day."

Why It Could Be Safer Than Statins

One of the main drawbacks of statins is that they can cause muscle pain, fatigue, or, in rare cases, liver or nerve problems. This leads some patients to stop statin treatment. The new DNA-based therapy avoids many of these issues because it does not directly interfere with the same cholesterol-making pathway that statins impact. Instead, it lets the liver boost its own cholesterol-clearing machinery in a more targeted way.

Researchers also point out that this strategy could be combined, if needed, with other cholesterol treatments for people with very high risk. The therapy might eventually require fewer doses than daily pills because it acts at the genetic level. This could improve long-term adherence and reduce the burden of side effects.

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