Innovative DNA Therapy Lowers Harmful Cholesterol Without Utilizing Statins

Scientists from the University of Barcelona and the University of Oregon have developed a novel DNA-based approach that specifically targets PCSK9, a protein associated with elevated LDL cholesterol levels and atherosclerosis. Their leading molecule, named HpE12, was shown to decrease PCSK9 protein levels by 87% in human liver cells and reduce cholesterol by 47% in genetically engineered mice after a single dose. This treatment employs uniquely crafted DNA structures known as polypurine hairpins to disrupt the gene's function, eliminating the need for statins. The effects diminished gradually over a two-week period, with the animals’ weight and liver function remaining stable. These findings, published in Biochemical Pharmacology, have yet to be evaluated in human subjects.
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