A new gene therapy treatment inhibiting the protein PCSK9 for the treatment of hypercholesterolemia.
Figure: This drug aims to inhibit a protein that increases the risk of heart disease by damaging arteries.
Previously, high cholesterol has been treated with statin medications, causing side effects of muscle weakness, increased blood sugar, and gastrointestinal issues. Recently, targeting the development of PCSK9 proteins has become increasing more prevalent. PCSK9 proteins cause harm by attaching themselves to LDL receptors and inhibit these proteins from removing excess cholesterol, eventually causing the buildup of LDL cholesterol.
The University of Barcelona has developed a new method of treating hypercholesterolemia with gene therapy. By utilizing polypurine hairpins, short molecules of DNA that bind to specific molecules, specific sites coding for PCSK9 are targeted and blocked from transcription. The subsequent increase in LDL receptors allows the body to pull cholesterol out of the blood stream and reduce plaque formation in the arteries. This new treatment is a safer way to regulate high cholesterol and risk of heart disease.
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