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DNA treatment cuts bad cholesterol by nearly 50% without statins

1 min
DNA treatment cuts bad cholesterol by nearly 50% without statins

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Researchers from the University of Barcelona and the University of Oregon have developed an experimental DNA-based treatment that reduces LDL cholesterol by nearly 50% without statins. The approach targets the PCSK9 gene using polypurine hairpins, increasing LDL receptors on cells. Findings were published in Biochemical Pharmacology.

Key Facts

  • The experimental treatment reduced LDL cholesterol by nearly 50% in preclinical studies.
  • The approach uses polypurine hairpins (PPRHs) to suppress the PCSK9 gene, increasing LDL receptors.
  • The research was led by Carles J. Ciudad and Verònica Noé of the University of Barcelona and Nathalie Pamir of the University of Oregon.
  • Findings were published in the journal Biochemical Pharmacology.

Mechanism of Action

PCSK9 reduces the number of LDL receptors on cell surfaces, leaving more LDL cholesterol in the bloodstream. The new strategy reduces expression of the PCSK9 gene itself, preventing the protein from being produced. Polypurine hairpins are short single-stranded DNA molecules designed to bind specific DNA or RNA sequences and interfere with gene transcription. Researchers tested two PPRHs, HpE9 and HpE12, both of which reduced PCSK9 RNA and protein while increasing LDLR levels.

Research Team and Funding

The study was led by Carles J. Ciudad and Verònica Noé from the University of Barcelona's Faculty of Pharmacy and Food Sciences and the Institute of Nanoscience and Nanotechnology (IN2UB). Nathalie Pamir of the University of Oregon in Portland collaborated on the research. The work was supported by the Spanish Ministry of Science, Innovation and Universities (MICINN) and the U.S. National Institutes of Health (NIH).

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