Home » Cholesterol, a gene causes the disease with the risk of heart attack and stroke: the study

Cholesterol, a gene causes the disease with the risk of heart attack and stroke: the study

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Cholesterol, a gene causes the disease with the risk of heart attack and stroke: the study

Italian Scientists Develop Breakthrough Therapy for Familial Hypercholesterolemia

A team of Italian scientists has made a groundbreaking discovery in the field of gene therapy, particularly in relation to a rare genetic condition known as familial hypercholesterolemia. This condition is characterized by dangerously high levels of cholesterol in the blood, leading to an increased risk of serious cardiovascular diseases such as heart attacks and strokes, even at a young age.

The gene responsible for regulating cholesterol levels, known as Pcsk9, has been the focus of the researchers’ work. Through a revolutionary epigenetic editing approach, the scientists have developed a method to effectively turn off the defective gene without altering the DNA itself. This marks the first time this advanced therapeutic strategy has been demonstrated in vivo, showing long-term effectiveness in a model organism.

The study, published in the prestigious journal Nature, has opened up new possibilities for treating not only familial hypercholesterolemia but also other diseases. By modulating the activation level of genes without changing their DNA sequence, this epigenetic editing technique offers a promising alternative to traditional genetic editing methods.

Lead researcher Angelo Lombardo, from the San Raffaele Telethon Institute for Gene Therapy in Milan, explains the significance of their findings. “This approach could revolutionize the treatment of hypercholesterolemia and potentially other diseases as well. It is a therapy that only needs to be administered once in a lifetime and has reversible effects, making it a safer and more efficient option.”

The successful results obtained by Lombardo’s team in animal models have paved the way for the development of drugs based on epigenetic silencing for hypercholesterolemia. The creation of a start-up, EpsilenBio, dedicated to further research and development in this field, signals a commitment to translating these exciting findings into tangible benefits for patients.

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With potential applications in various other diseases, including hepatitis B and central nervous system disorders, this groundbreaking therapy offers hope for a new era of personalized medicine. The future of gene therapy looks brighter than ever, thanks to the innovative work of these Italian scientists.

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