New DNA-Based Therapy Shows Promise in Reducing Cholesterol Levels
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Researchers have developed a DNA-based method to control cholesterol by targeting the PCSK9 protein, which regulates LDL cholesterol levels. In tests on cells and mice, the therapy reduced PCSK9 protein levels by up to 87% and cholesterol levels by 47%. This could lead to a new approach for treating high cholesterol and reducing heart disease risk.
Facts First
- Researchers developed DNA molecules called polypurine hairpins (PPRHs) to block production of the cholesterol-regulating PCSK9 protein.
- A specific PPRH, HpE12, reduced PCSK9 RNA by 74% and protein by 87% in laboratory liver cells.
- A single injection of HpE12 in mice reduced plasma PCSK9 levels by 50% and cholesterol levels by 47% within three days.
- The method differs from existing treatments like siRNA (Inclisiran) and monoclonal antibodies (evolocumab, alirocumab).
- The study was conducted by teams from the University of Barcelona and the University of Oregon and published in Biochemical Pharmacology.
What Happened
Researchers from the University of Barcelona and the University of Oregon have developed a new method to control cholesterol levels. They used specialized DNA-based molecules called polypurine hairpins (PPRHs) to target the PCSK9 protein, which regulates low-density lipoprotein cholesterol (LDL-C) by limiting cells' ability to remove it from the blood. Two specific PPRHs, named HpE9 and HpE12, were designed to bind to sequences in the PCSK9 gene, blocking its transcription. The research was tested in laboratory-grown liver cells and in transgenic mice carrying the human PCSK9 gene. Professor Verònica Noé reported that HpE12 decreased PCSK9 RNA levels by 74% and protein levels by 87% in cells. In mice, a single injection of HpE12 reduced plasma PCSK9 levels by 50% and cholesterol levels by 47% on the third day.
Why this Matters to You
High cholesterol (hypercholesterolemia) damages arteries and increases your risk of heart disease. This new research could eventually lead to a more effective treatment option for managing your cholesterol levels. If developed into a therapy, it might offer a different mechanism compared to existing drugs, potentially providing a new choice for patients who do not respond optimally to current treatments. The significant reductions observed in early tests suggest this approach could be a powerful tool in preventing cardiovascular disease.
What's Next
The research, published in the journal Biochemical Pharmacology, represents an early but promising step. Further studies will be needed to confirm safety and efficacy in humans before this method could become a available treatment. The development may progress alongside or offer an alternative to existing PCSK9-targeting therapies like Inclisiran and monoclonal antibodies.