Innovative Therapeutic Approach to Lower Cholesterol Levels
Researchers have developed a new gene therapy using polypurine hairpins to inhibit PCSK9, offering a promising and safer strategy to lower LDL cholesterol and combat cardiovascular disease.
A recent study led by researchers from the University of Barcelona and the University of Oregon introduces a groundbreaking method to regulate blood cholesterol, offering new hope in the fight against cardiovascular diseases. Hypercholesterolemia, characterized by excessive cholesterol in the blood, can lead to serious arterial damage and atherosclerosis—the buildup of lipid plaques in artery walls. The newly developed strategy focuses on inhibiting the expression of PCSK9, a protein that plays a pivotal role in controlling low-density lipoprotein cholesterol (LDL-C) levels.
The research utilizes polypurine hairpins (PPRHs), which are specially designed molecules that can selectively interfere with gene transcription. By targeting the PCSK9 gene, PPRHs prevent its expression, resulting in increased LDL receptor availability on cell surfaces. This enhances the uptake of cholesterol into cells and reduces its circulation in the blood, effectively decreasing the risk of plaque formation.
The study demonstrates that PPRHs, particularly HpE9 and HpE12, significantly lower PCSK9 RNA and protein levels in cell models and transgenic mice. In vivo experiments showed that a single injection of HpE12 reduced plasma PCSK9 by 50% and cholesterol levels by 47% within three days. This approach was validated in HepG2 cells and transgenic mice expressing human PCSK9, confirming its potential as an effective therapeutic tool.
Currently, therapies targeting PCSK9 include monoclonal antibodies like evolocumab and alirocumab, as well as gene silencing techniques such as siRNAs and CRISPR-based methods. However, PPRHs offer several advantages, including lower production costs, stability, and minimal immunogenicity, without the side effects associated with statins like myopathies.
Experts highlight that this novel gene therapy approach could serve as a cost-effective and safer alternative to existing treatments, broadening options for patients with hypercholesterolemia and reducing the incidence of cardiovascular complications.
This innovative strategy underscores the potential of oligonucleotide-based therapies in managing lipid disorders and opens new avenues for personalized medicine in cardiovascular health.
Source: https://medicalxpress.com/news/2025-06-therapeutic-strategy-cholesterol.html
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