Home » The Pcsk9 gene in the sights of scientists against hypercholesterolemia

The Pcsk9 gene in the sights of scientists against hypercholesterolemia

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The Pcsk9 gene in the sights of scientists against hypercholesterolemia

Revolutionary treatment against hypercholesterolemia discovered: a novel approach that turns off the culprit gene.

A team of Italian researchers recently conducted a groundbreaking study on gene Pcsk9involved in the regulation of cholesterol. The result? A new technique to turn off the gene without modifying the DNA, with potential revolutionary applications in the treatment of cardiovascular and cerebrovascular diseases.

Published in the prestigious journal Nature, the study demonstrates the long-term effectiveness of a innovative epigenetic approach, designed to modulate gene expression without altering the DNA sequence. This strategy, conceived by a team led by Professor Angelo Lombardoat the San Raffaele Telethon Institute for Gene Therapy (Sr. Tiget) of Milan, could open new horizons in the treatment of a series of genetic pathologies, includingfamilial hypercholesterolemia.


The technology, which involves creating “editor” molecules that can recognize and switch off the faulty gene, has already shown promising results in in vitro experiments and animal models. By adopting lipid nanoparticles similar to those used in mRNA-based vaccines, Lombardo’s team was able to successfully silence the Pcsk9 gene in mouse models.

The potential applications of the technology are not just limited to hypercholesterolemia, as the epigenetic approach could be adapted to address other pathologies, such ashepatitis B and even central nervous system disorders. However, to bring these advances from the laboratory to the bedside, it is essential to continue research and testing.

Thanks to partnerships with the Telethon Foundation and the San Raffaele hospital, Professor Lombardo and Professor Luigi Naldini created EpsilenBio, a start-up dedicated to the development of this promising technology. The company was recently acquired by Chroma Medicine Inc.one of the leading companies in the epigenetic silencing sector, confirming the interest and confidence in the potential of this innovative gene therapy.

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This pioneering study, with its innovative approach and promising applications, marks a significant milestone in the fight against genetic diseases and offers new hope for patients living with such conditions.

Nurse Times editorial team

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