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High cholesterol gene turned off, success in tests on mice

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High cholesterol gene turned off, success in tests on mice

Italian researchers have achieved a groundbreaking success in the fight against high cholesterol, a common risk factor for heart disease. Using a new technique, scientists were able to ‘turn off’ the gene responsible for high cholesterol in mice without modifying the DNA sequence, resulting in a long-lasting effect that remained active for about a year.

The study, published in the journal Nature, was led by the San Raffaele Telethon Institute for Gene Therapy in Milan, in collaboration with the University of Eastern Piedmont and other research institutions. This innovative approach to gene therapy could have far-reaching implications for the treatment of other diseases as well.

The researchers focused their efforts on the Pcsk9 gene, which plays a key role in regulating cholesterol levels in the blood. By targeting this gene, which is linked to familial hypercholesterolemia, a rare genetic condition that increases the risk of heart disease at a young age, the team was able to achieve significant results.

Rather than directly modifying the DNA sequence, the researchers developed molecules designed to recognize and switch off the Pcsk9 gene by modifying the epigenome, the set of molecules that control gene expression without altering the DNA. These molecules were then encapsulated in nanoparticles and delivered to the liver, where they successfully silenced the gene in mice with just one administration.

The success of this study opens up new possibilities for the development of novel therapies for hypercholesterolemia and other liver-related disorders. By demonstrating the effectiveness of this technique in live animals, the researchers have paved the way for potential treatments for a wide range of conditions, including hepatitis B and central nervous system disorders.


This innovative approach to gene therapy offers potential advantages over traditional methods, as it can be administered once in a lifetime, does not carry the risk of DNA mutations, and is potentially reversible. The promising results of this study highlight the potential of epigenetic silencing as a valuable tool in the fight against various diseases, bringing hope for improved treatments in the future.

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